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test-1501
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Endocrinology
ENDO
[ ". Her menstrual cycle was regular and she was menstruating one day after admission." ]
test-1502
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Genitourinary-System
GU
[]
test-1503
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Respiratory-System
RESP
[]
test-1504
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Musculoskeletal-System
MSK
[]
test-1505
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Eyes-Ears-Nose-Throat
EENT
[]
test-1506
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Dermatology
DERM
[]
test-1507
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Pregnancy
Pregnancy
[]
test-1508
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Lymphatic-System
LYMPH
[]
test-1509
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Age-at-Presentation
Age (at case presentation)
[ "a 24 year - old", "24 year - old-" ]
test-1510
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Age-of-Onset
Age (of onset)
[]
test-1511
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
Confirmed-Diagnosis-IEM
Confirmed_Diagnosis(IEM)
[ "acute intermittent porphyria", "The diagnosis of AIP was based on the presence of high PBG in the urine ( test was done at another center ) ." ]
test-1512
5633257
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
{'Case presentation': 'We present a 24 year-old woman who developed acute intermittent porphyria five days after right hemi-colectomy. Her presentation included neuro-visceral and psychiatric manifestations, and severe hyponatremia. She received critical care symptomatic management including mechanical ventilation. The diagnosis was based on a positive urine test for porphobilinogen and confirmed by the presence of a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). This work has been reported in line with the SCARE criteria . A 24 year-old- single female patient was admitted because of right lower abdominal pain and distension for 2 weeks. The pain was colicky, episodic, with no precipitating factors and only relieved after bowel motion. She had no constitutional symptoms of chronic illness. She was not on any medications and her social history was unremarkable. Her menstrual cycle was regular and she was menstruating one day after admission. Physical examination revealed a thin patient in mild pain distress with normal vital signs. Her abdomen was soft and lax with normal bowel sounds. Routine laboratory tests including CBC and renal and liver function tests were normal. Serum transaminases remained normal during her hospital stay. Aspartate aminotransferase (AST, SGOT) range was 18–33 U/L (normal range: 15–37 U/L). Alanine aminotransferase (ALT, SGPT) range was 25–51 U/L (normal range: 14–63 U/L). On admission, level of serum sodium (Na) was normal: 138 mEq/L (normal range 135–145 mEq/L) and remained normal (range: 135–139 mEq/L) until the 5th postoperative day (see below). Plain abdominal X-ray and abdominal computed tomography (CT) showed a distended cecum located in the pelvis with fecal impaction; the rest of the viscera were unremarkable. These findings were consistent with mobile cecum syndrome. The patient was scheduled for laparoscopy and possible laparotomy. The only abnormality during laparoscopy was that the cecum and ascending colon were not attached to posterior abdominal wall. The procedure was converted to laparotomy. Right hemi-colectomy was performed. She was kept on epidural analgesia, intravenous (IV) fluids and nil by mouth for four days and had a smooth post-operative course. After surgery, she received intravenous (IV) 5% dextrose/0.9% NaC) three liters/day (her weight was 48 kg). In addition, her pain was controlled by epidural analgesia of lidocaine and fentanyl. The histology revealed normal colon. On the 5th postoperative day, oral intake was resumed and epidural analgesia was discontinued. Eight hours later, she had a sudden deterioration of consciousness level and became unresponsive with a Glasgow Coma Scale of 7/15. Her pupils were dilated and had a sluggish reaction to light. The patient was immediately intubated, mechanically ventilated, resuscitated, and admitted to the intensive care unit (ICU). Blood work up revealed severe hyponatremia; serum Na: 107 mEq/l with normal renal and liver function tests. Our differential diagnoses for the severe hyponatremia included drug intoxicity, encephalitis, and salt losing nephropathy. Consultations to neurology and psychiatry were made and they advised brain CT scan and magnetic resonance imaging (MRI), and cerebrospinal fluid analysis. These tests and septic work-up were normal. She was extubated two days later. At this stage, she started to complain of acute abdominal pain and developed altered mental status, personality changes, visual and auditory hallucinations with euphoric and aggression episodes despite correction of serum Na. The presence of abdominal pain, neurological and psychiatric symptoms and unexplained severe hyponatremia raised the suspicion of AIP. The diagnosis of AIP was based on the presence of high PBG in the urine (test was done at another center). At no time, her urine was dark. In retrospect, the family history showed that two of her cousins (from mother side) had AIP. The patient condition improved markedly after proper pain control and high carbohydrate intake. We referred her to a higher center where she was found to have a heterozygous mutation in the hydroxyrmethylbilane synthase ( HMBS ) gene (c.760delC p Leu254). There, she received premenstrual regime of IV hematin (heme arginate) 4 mg/Kg body weight/day for three days. She had four recurrent milder attacks in the form of abdominal and back pain and confusion and was treated with similar regime of hematin. Thereafter, she was kept on once monthly prophylactic IV hematin 4 mg/Kg body weight/day for three days. Now, one and a half years after discharge from our service, she is doing fine and on no hematin treatment.'}
IEM-Treatment
IEM_Treatment
[ "high carbohydrate intake .", "regime of IV hematin ( heme arginate ) 4 mg / Kg body weight / day for three days .", "prophylactic IV hematin 4 mg / Kg body weight / day for three days" ]
test-1513
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Vitals-and-Hematology
Vitals_Hema
[ "polycythemia ( hemoglobin : 18.0 g / dL; red blood count : 627 × 10 4; hematocrit : 53.1 %", "height : 165 cm; weight : 79 kg; Body Mass Index : 29.0 kg / m²", "polycythemia also improved to a Hb level of 15.2 g / dL.", "height : 1.58 m; weight : 65 kg; body mass index : 23.9 kg / m²" ]
test-1514
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Gastrointestinal-System
GI
[]
test-1515
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Patient-History
History
[ "A 41 year old man was followed at the local clinic for polycythemia ( hemoglobin : 18.0 g / dL; red blood count : 627 × 10 4; hematocrit : 53.1 % ) and hypertension", "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility", "female offspring ( 3650 g ) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement." ]
test-1516
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Neurology
Neuro
[]
test-1517
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Laboratory-and-Imaging
Lab_Image
[ "polycythemia ( hemoglobin : 18.0 g / dL; red blood count : 627 × 10 4; hematocrit : 53.1 % )", "An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands", "The laboratory tests revealed an extremely high level of ACTH ( 316.4 pg / mL ) and a relatively low level of cortisol ( 6.8 μg / dL ). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary – adrenal hormones, and high level of serum 17‐OHP ( 62.7 ng / mL ) indicated the possibility of CAH.", "The abnormality in the pituitary – gonadal hormonal status ( FSH : < 0.1 IU / mL; LH : < 0.1 IU / mL; testosterone : 10.9 ng / mL; estradiol : 29 pg / mL ) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan", "All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 ( Figure 6 A ). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation", "A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes ( Figure 2 A ). The semen analysis revealed oligoasthenozoospermia ( volume : 2.6 mL; concentration : 7 × 10 6 /mL; motility : 14 % ). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone ( FSH ) of 2.7 mIU / mL, luteinizing hormone ( LH ) of 1.3 mIU / mL, testosterone of 4.3 ng / mL, and estradiol of 13 pg / mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging ( MRI ) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image ( Figure 3 ). An enhanced computed tomography ( CT ) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes ( Figure 4 A, B ). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors.", "In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high ( 69.3 pg / mL and 112 ng / mL, respectively ). Genetic diagnosis by polymerase chain reaction ( PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation ( I‐172N mutation" ]
test-1518
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Cardiovascular-System
CVS
[ "hypertension" ]
test-1519
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Endocrinology
ENDO
[]
test-1520
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Genitourinary-System
GU
[ "Bilateral testicular tumors were identified in a scrotal ultrasound echo scan", "both testes had become slightly atrophied ( right : 14 mL; left : 12 mL ) ( performed with a Prader orchidmeter ) and no induration was palpated", "serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 ( Figure 6 A ). The semen analysis showed azoospermia", "Sperm emerged ( concentration : 3.4 × 10 6 /mL; motility : 26 % )", "size reduction in the adrenal hyperplasia and TART", "infertility", "Both testes had become atrophied ( 9 mL ) ( performed with a Prader orchidmeter ) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes ( Figure 2 A ). The semen analysis revealed oligoasthenozoospermia ( volume : 2.6 mL; concentration : 7 × 10 6 /mL; motility : 14 % )", "irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image", "no finding in the testes", "Bilateral testicular atrophy", "semen analysis revealed a significant change ( concentration : 34 × 10 6 /mL; motility : 59 % ), along with normalization of the testicular size ( right : 14 mL; left : 16 mL ). The size reduction of the TART was identified by ultrasound" ]
test-1521
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Respiratory-System
RESP
[]
test-1522
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Musculoskeletal-System
MSK
[]
test-1523
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Eyes-Ears-Nose-Throat
EENT
[]
test-1524
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Dermatology
DERM
[]
test-1525
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Pregnancy
Pregnancy
[]
test-1526
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Lymphatic-System
LYMPH
[]
test-1527
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Age-at-Presentation
Age (at case presentation)
[ "41 year old" ]
test-1528
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Age-of-Onset
Age (of onset)
[]
test-1529
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
Confirmed-Diagnosis-IEM
Confirmed_Diagnosis(IEM)
[ "Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility , accompanied by congenital adrenal hyperplasia ( CAH )" ]
test-1530
5768970
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
{'Cases': 'Testicular adrenal rest tumor ( TART ) is one of the possible causes of male infertility, accompanied by congenital adrenal hyperplasia ( CAH ). Here are reported two cases of TART s that were referred to Kobe City Medical Center West Hospital for the treatment of infertility and testicular tumors.', 'Case 2': 'A 41 year old man was followed at the local clinic for polycythemia (hemoglobin: 18.0 g/dL; red blood count: 627 × 10 4 ; hematocrit: 53.1%) and hypertension. An abdominal CT scan for the examination of these conditions revealed the enlargement of the bilateral adrenal glands (Figure 5 ). The patient was referred to endocrinologists in the hospital for further investigation. The patient was seen to have obesity (height: 165 cm; weight: 79 kg; Body Mass Index: 29.0 kg/m²). The laboratory tests revealed an extremely high level of ACTH (316.4 pg/mL) and a relatively low level of cortisol (6.8 μg/dL). Although the patient did not have any family history of CAH, the CT findings regarding the adrenal glands, unbalanced pituitary–adrenal hormones, and high level of serum 17‐OHP (62.7 ng/mL) indicated the possibility of CAH. The difference from case 1 is that the first examination was made by endocrinologists, not urologists. The differential diagnosis was made before a testicular examination. The abnormality in the pituitary–gonadal hormonal status (FSH: <0.1 IU/mL; LH: <0.1 IU/mL; testosterone: 10.9 ng/mL; estradiol: 29 pg/mL) indicated the excessive secretion of adrenal androgens. Bilateral testicular tumors were identified in a scrotal ultrasound echo scan and then a urological consultation and several additional examinations were offered in order to rule out a germ cell tumor. At the physical examination, both testes had become slightly atrophied (right: 14 mL; left: 12 mL) (performed with a Prader orchidmeter) and no induration was palpated. All the serum markers for testicular tumors were within the normal range and the testicular MRI showed the same sign as case 1 (Figure 6 A). The semen analysis showed azoospermia. A genetic diagnosis by PCR‐direct sequencing proved CAH due to 21‐hydroxylase deficiency due to CYP21A2 gene mutation. After 6 months of low‐dose, daily oral glucocorticoid therapy (0.5 mg/d), the serum FSH, LH, testosterone, and estradiol levels returned to normal levels (9.2 IU/mL, 1.8 IU/mL, 0.8 ng/mL, and <10 pg/mL, respectively). The polycythemia also improved to a Hb level of 15.2 g/dL. Sperm emerged (concentration: 3.4 × 10 6 /mL; motility: 26%), along with a size reduction in the adrenal hyperplasia and TART (Figure 6 B).', 'Case 1': "A 41 year old man was referred to Kobe City Medical Center West Hospital, Kobe City, Japan, because of 2 years of infertility. At the physical examination, the patient's physical constitution was as follows (height: 1.58 m; weight: 65 kg; body mass index: 23.9 kg/m²). Both testes had become atrophied (9 mL) (performed with a Prader orchidmeter) and pea‐sized indurations were felt around the epididymal head in both testes. A scrotal ultrasound examination confirmed the presence of bilateral heteroechogenic, hypovascularized masses near the testes (Figure 2 A). The semen analysis revealed oligoasthenozoospermia (volume: 2.6 mL; concentration: 7 × 10 6 /mL; motility: 14%). The hormonal examination revealed a normal hormonal status of serum follicle‐stimulating hormone (FSH) of 2.7 mIU/mL, luteinizing hormone (LH) of 1.3 mIU/mL, testosterone of 4.3 ng/mL, and estradiol of 13 pg/mL. All the serum markers for testicular tumors, including human chorionic gonadotropin, alpha‐fetoprotein, and lactate dehydrogenase, were within the normal range. The pelvic magnetic resonance imaging (MRI) scan showed irregular, margin, heterogeneous low‐signal‐intensity tumors that were surrounded by high‐signal normal testicular tissue in the T2‐weighted image (Figure 3 ). An enhanced computed tomography (CT) scan revealed diffuse, irregular enlargement of both adrenal glands, with no finding in the testes (Figure 4 A,B). Bilateral testicular atrophy and bilateral enlargement of the adrenal glands without any other metastases did not seem to be typical for malignant testicular tumors. At this point, adrenal gland disease was suspected and an endocrinologist was consulted for further examination. In the endocrinological examination, both the serum ACTH and 17‐OHP levels were extremely high (69.3 pg/mL and 112 ng/mL, respectively). Genetic diagnosis by polymerase chain reaction (PCR)‐direct sequencing confirmed the 21‐hydroxylase deficiency due to CYP21A2 gene mutation (I‐172N mutation) and a low‐dose, oral glucocorticoid of 0.5 mg/d was started under the diagnosis of CAH. After 1 month of glucocorticoid therapy of 0.5 mg/d, the semen analysis revealed a significant change (concentration: 34 × 10 6 /mL; motility: 59%), along with normalization of the testicular size (right: 14 mL; left: 16 mL). The size reduction of the TART was identified by ultrasound (Figure 2 B). The hormonal status had not changed significantly from the pretreatment status (serum FSH: 2.8 mIU/mL; LH: 1.6 mIU/mL; testosterone: 3.2 ng/mL; estradiol: 18 pg/mL). After 2 years of glucocorticoid administration, the clinical pregnancy of his wife was established by using in vitro fertilization (IVF), resulting in the delivery of a female offspring (3650 g) who was confirmed as negative for CAH by serum 17‐OHP and 21‐deoxycortisol measurement."}
IEM-Treatment
IEM_Treatment
[ "After 6 months of low‐dose , daily oral glucocorticoid therapy ( 0.5 mg / d ) , the serum FSH , LH , testosterone , and estradiol levels returned to normal levels ( 9.2 IU / mL , 1.8 IU / mL , 0.8 ng / mL , and < 10 pg / mL , respectively ) . The polycythemia also improved to a Hb level of 15.2 g / dL.", "a low‐dose , oral glucocorticoid of 0.5 mg / d was started under the diagnosis of CAH" ]
test-1531
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Vitals-and-Hematology
Vitals_Hema
[]
test-1532
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Gastrointestinal-System
GI
[]
test-1533
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Patient-History
History
[ "In 1989, a 21 - year - old male experienced visual loss after diving from a diving board", "Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye" ]
test-1534
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Neurology
Neuro
[]
test-1535
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Laboratory-and-Imaging
Lab_Image
[ "Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes", "Analysis showed that our patient was homozygous for the R1141X - mutation in the ABCC6 - gene" ]
test-1536
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Cardiovascular-System
CVS
[]
test-1537
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Endocrinology
ENDO
[]
test-1538
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Genitourinary-System
GU
[]
test-1539
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Respiratory-System
RESP
[]
test-1540
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Musculoskeletal-System
MSK
[]
test-1541
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Eyes-Ears-Nose-Throat
EENT
[ "visual loss after diving from a diving board", "bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch ’s membrane were identified. The macula was not affected and vision returned to normal in both eyes", "recent visual loss in the left eye", "Visual acuity ( VA ) was 20/25 in the right eye ( RE ) and 20/40 in the left eye ( LE )", "Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema", "observed in both eyes angioid streaks, peau d’orange and a few comet tails", "sequellae of diving induced breaks of Bruchs ’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks", "growth of the choroidal neovascularization ( CNV ) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye", "VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV" ]
test-1542
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Dermatology
DERM
[ "no typical plucked - chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes" ]
test-1543
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Pregnancy
Pregnancy
[]
test-1544
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Lymphatic-System
LYMPH
[]
test-1545
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Age-at-Presentation
Age (at case presentation)
[ "21 - year - old" ]
test-1546
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Age-of-Onset
Age (of onset)
[]
test-1547
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
Confirmed-Diagnosis-IEM
Confirmed_Diagnosis(IEM)
[ "PXE" ]
test-1548
5015608
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
{'Case report': 'In 1989, a 21-year-old male experienced visual loss after diving from a diving board. Ocular examination was performed the same day, and revealed bilateral subretinal hemorrhages and angioid streaks. After clearing of the hemorrhages, more breaks of Bruch’s membrane were identified. The macula was not affected and vision returned to normal in both eyes. Twenty years later, in April 2009, he presented again because of recent visual loss in the left eye. Visual acuity (VA) was 20/25 in the right eye (RE) and 20/40 in the left eye (LE). Clinical examination, FA, and OCT revealed active choroidal neovascularisation nasal to the fovea with leakage and edema. One month after a first intravitreal injection of bevacizumab, the edema had resolved and vision improved. Close follow-up with consideration of additional anti-VEGF injections was adviced. Moreover, we observed in both eyes angioid streaks, peau d’orange and a few comet tails, and made a tentative diagnosis of PXE. We identified the sequellae of diving induced breaks of Bruchs’ membrane with linear vertical break formation, different from the irregular course and spiderweb configuration of the angioid streaks. During the following months a total of 8 injections of bevacizumab were administred on the LE and despite this treatment growth of the choroidal neovascularization (CNV) was observed with further deterioration of vision. In January 2010, active CNV was also identified in the right eye and treatment with bevacizumab intravitreal injections was initiated. At last examination, in April 2011, VA was 20/60 in the right eye and 20/250 in the left eye, with in the left eye a large subfoveal CNV expanding on both sides of the fovea, and in the RE a smaller and mildly active subfoveal CNV requiring further treatment (Figure 1 (Fig. 1), Figure 2 (Fig. 2), Figure 3 (Fig. 3), Figure 4 (Fig. 4), Figure 5 (Fig. 5), Figure 6 (Fig. 6), Figure 7 (Fig. 7), Figure 8 (Fig. 8), Figure 9 (Fig. 9), Figure 10 (Fig. 10) ). With the aim to confirm PXE, the patient was referred for dermatologic examination and biopsy. At age 43, there were no typical plucked-chicken appearance, no papules, macules or other skin lesions. Three biopsies from the flexural skin of the axilla and the neck did not reveal PXE related changes. Subsequently, to further exclude or confirm PXE, a blood sample for genetic analysis was taken. Analysis showed that our patient was homozygous for the R1141X-mutation in the ABCC6-gene. The diagnosis of PXE was withheld, discussed with the patient, and shared with his treating generalist to allow optimal further follow-up of the condition.'}
IEM-Treatment
IEM_Treatment
[]
test-1549
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Vitals-and-Hematology
Vitals_Hema
[ "height and weight were 86 cm and 12.5 kg, respectively ( both were below the 3rd percentile, according to China 's 1995 urban 0–18 year - old male height percentiles scale", "height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively", "his height and weight reached 121 cm and 22 kg" ]
test-1550
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Gastrointestinal-System
GI
[ "showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting", "short stature and excessive thinness" ]
test-1551
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Patient-History
History
[ "The patient was born at 36 +4 weeks ’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness", "At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance", "A Chinese boy initially presented with severe stunting and partial growth hormone deficiency ( PGHD ) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later ( at 10 years old ), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy." ]
test-1552
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Neurology
Neuro
[ "developmental milestones became delayed", "began to walk at 1 year and 6 months and his walk was slower than normal and clumsy", "A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings", "a single episode of generalized tonic seizure", "previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma", "gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance", "muscle strength of the low extremities was grade 4 ( Oxford Scale ), and he showed increased distal muscle tone than normal, hyper - reflexia, and a negative Babinski sign", "normal, as were the results of cranial MRI and electroencephalography", "peripheral nerve examination revealed reduced compound muscle action potential ( CMAP ) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged", "A muscle biopsy revealed neurogenic lesion", "His intelligence quotient was lower than normal, at 80", "mental state and vomiting had clearly improved", "spastic diplegia symptoms had not improved", "spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy" ]
test-1553
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Laboratory-and-Imaging
Lab_Image
[ "Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.", "Laboratory tests revealed retardation of bone age ( equivalent to a 1 year old ) and mildly elevated total bilirubin ( 29.4 μmol / L, normal range : 2–24 μmol / L ), direct bilirubin ( 10.4 μmol / L, normal range : 0–7 μmol / L ), and indirect bilirubin levels ( 18.6 μmol / L, normal range : 1.7–17 μmol / L ) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal ( 3.52 mmol / L, normal range : 3.9–6 ), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone ( 0 minute : 0.76 ng / mL; 30 minutes : 4.5 ng / mL; 60 minutes : 6.3 ng / mL; 90 minutes : 4.1 ng / mL; and 120 minutes : 1.5 ng / mL ). An MRI revealed a normal pituitary gland, and thyroid function was normal.", "Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase ( 50.9 IU / L, normal range : 0–50 IU / L ) and alanine transaminase ( 52 IU / L, normal range : 0–45 IU / L ), as well as mildly elevated total bilirubin ( 47.7 μmol / L, normal range : 0–25 μmol / L ), direct bilirubin ( 16.4 μmol / L, normal range : 0–6.8 μmol / L ), and indirect bilirubin levels ( 31.3 μmol / L, normal range : 0–25 μmol / L ). His creatine kinase ( 564.4 IU / L, normal range : 55–190 IU / L ) and blood ammonia levels ( 62.3 mmol / L, normal range : 9–30 mmol / L ) were slightly elevated. A urine occult blood test was positive ( + + ). Red blood cells ( 3–4 / HP, normal range : 0–3 / HP ) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography.", "A muscle biopsy revealed neurogenic lesion ( Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 ( c.32 T > C )", "Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM ( normal range : 10–50 μM ) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal" ]
test-1554
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Cardiovascular-System
CVS
[]
test-1555
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Endocrinology
ENDO
[ "retardation of bone age ( equivalent to a 1 year old )", "Blood glucose was slightly lower than normal ( 3.52 mmol / L, normal range : 3.9–6", "A growth hormone excitement test revealed low levels of growth hormone ( 0 minute : 0.76 ng / mL; 30 minutes : 4.5 ng / mL; 60 minutes : 6.3 ng / mL; 90 minutes : 4.1 ng / mL; and 120 minutes : 1.5 ng / mL ). An MRI revealed a normal pituitary gland, and thyroid function was normal", "PGHD", "severe stunting and partial growth hormone deficiency ( PGHD )" ]
test-1556
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Genitourinary-System
GU
[]
test-1557
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Respiratory-System
RESP
[]
test-1558
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Musculoskeletal-System
MSK
[ "muscle strength of the low extremities was grade 4 ( Oxford Scale )", "His creatine kinase ( 564.4 IU / L, normal range : 55–190 IU / L ) and blood ammonia levels ( 62.3 mmol / L, normal range : 9–30 mmol / L ) were slightly elevated" ]
test-1559
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Eyes-Ears-Nose-Throat
EENT
[]
test-1560
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Dermatology
DERM
[]
test-1561
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Pregnancy
Pregnancy
[ "born at 36 +4 weeks ’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia." ]
test-1562
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Lymphatic-System
LYMPH
[]
test-1563
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Age-at-Presentation
Age (at case presentation)
[ "10 years old" ]
test-1564
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Age-of-Onset
Age (of onset)
[ "1 year of age" ]
test-1565
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
Confirmed-Diagnosis-IEM
Confirmed_Diagnosis(IEM)
[ "argininemia with homozygous mutation ( c.32T > C ) of the ARG1 gene", "argininemia" ]
test-1566
5839826
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
{'Diagnoses:': 'Ultimately, the patient was diagnosed with argininemia with homozygous mutation (c.32T>C) of the ARG1 gene at 10 years old. Blood tests showed mildly elevated blood ammonia and creatine kinase, and persistently elevated bilirubin.', 'Case report': "The patient was born at 36 +4 weeks’ gestation with a birth weight of 2500 g. Three days after birth, he was hospitalized for several days due to transient hypoglycemia. His developmental milestones were unremarkable before 1 year of age. Following this, his developmental milestones became delayed, for example, the boy began to walk at 1 year and 6 months and his walk was slower than normal and clumsy. There was no family history of severe childhood illness, death, or stunting. The boy showed a preference for carbohydrate intake from infancy, with a clear aversion to animal proteins and was prone to vomiting. The boy has a sister showing normal development. The boy presented to our child health clinic at age 3 years and 7 months because of his short stature and excessive thinness. At this time, his height and weight were 86 cm and 12.5 kg, respectively (both were below the 3rd percentile, according to China's 1995 urban 0–18 year-old male height percentiles scale). A neurologic examination focusing on the motor and sensory nervous system and cerebellar function produced unremarkable findings. Laboratory tests revealed retardation of bone age (equivalent to a 1 year old) and mildly elevated total bilirubin (29.4 μmol/L, normal range: 2–24 μmol/L), direct bilirubin (10.4 μmol/L, normal range: 0–7 μmol/L), and indirect bilirubin levels (18.6 μmol/L, normal range: 1.7–17 μmol/L) but normal hepatic enzyme levels. Blood glucose was slightly lower than normal (3.52 mmol/L, normal range: 3.9–6), whereas his electrolytes were normal. Screening tests for hepatitis B and A were negative. A growth hormone excitement test revealed low levels of growth hormone (0 minute: 0.76 ng/mL; 30 minutes: 4.5 ng/mL; 60 minutes: 6.3 ng/mL; 90 minutes: 4.1 ng/mL; and 120 minutes: 1.5 ng/mL). An MRI revealed a normal pituitary gland, and thyroid function was normal. The boy was diagnosed with PGHD and managed accordingly, being treated with growth hormone for about 1 month. No treatment effect was evident. However, he received no follow-up until 10 years of age. At 10 years old, the boy presented to our pediatric neurological clinic because of a single episode of generalized tonic seizure. The boy had previously had a seizure at 8 years old when receiving tetanus antitoxin treatment after head trauma. During clinic counseling, one of his parents noted that the patient had gait disturbance for almost 1 year, attributed to a persistent tightening of both knee joints. The boy could not jump and easily fell when he ran and walked. The boy, who was in primary school, also showed poor academic performance. At this time, his height and weight were still below the 3rd percentile, at 116 cm and 20.1 kg, respectively. He exhibited no organomegaly. His muscle strength of the low extremities was grade 4 (Oxford Scale), and he showed increased distal muscle tone than normal, hyper-reflexia, and a negative Babinski sign. Laboratory tests revealed mildly elevated hepatic enzyme levels, including aspartate transaminase (50.9 IU/L, normal range: 0–50 IU/L) and alanine transaminase (52 IU/L, normal range: 0–45 IU/L), as well as mildly elevated total bilirubin (47.7 μmol/L, normal range: 0–25 μmol/L), direct bilirubin (16.4 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (31.3 μmol/L, normal range: 0–25 μmol/L). His creatine kinase (564.4 IU/L, normal range: 55–190 IU/L) and blood ammonia levels (62.3 mmol/L, normal range: 9–30 mmol/L) were slightly elevated. A urine occult blood test was positive (++). Red blood cells (3–4/HP, normal range: 0–3/HP) were evident in a urine sediment microscopy. Both renal ultrasound and renal function were normal, as were the results of cranial MRI and electroencephalography. A peripheral nerve examination revealed reduced compound muscle action potential (CMAP) of the right peroneal nerve and bilateral tibial nerve, while the right peroneal nerve distal latency was suspected to be prolonged. A muscle biopsy revealed neurogenic lesion (Fig. 1 ). A gene test was performed and the results demonstrated that he carried compound homozygote mutations of ARG1 (c.32T > C) (Fig. 2 ). Lee et al and Wu et al reported the mutation, which can lead to argininemia, in South Korea and Chinese populations in 2011 and 2015. Subsequently, his blood and urine amino acid levels were analyzed, revealing a blood arginine concentration of 108.42 μM (normal range: 10–50 μM) as well as elevated urinary excretion of orotic acid; however, his blood citrulline, glutamine, glutamate, and alanine concentrations were normal. His intelligence quotient was lower than normal, at 80. The boy subsequently diagnosed with argininemia. Following the diagnosis, we recommended that the patient begins a low-protein diet (0.8 g/kg/day) to decrease his blood arginine levels, as well as start taking citrulline (150–200 mg/kg/day). His mental state and vomiting had clearly improved after 3 months of this treatment. Laboratory tests conducted again at a local hospital which the following values were obtained (note that the normal ranges used at this hospital slightly differ from those used at our hospital): aspartate transaminase (45 IU/L, normal range: 0–37 IU/L), alanine transaminase (46.1 IU/L, normal range: 0–40 IU/L), total bilirubin (31.9 μmol/L, normal range: 3.4–20.5 μmol/L), direct bilirubin (14.3 μmol/L, normal range: 0–6.8 μmol/L), and indirect bilirubin levels (17.6 μmol/L, normal range: 0–18 μmol/L). His urine occult blood test was positive (+). No red blood cells were evident in his urine sediment microscopy. After 9 months treatment, his height and weight reached 121 cm and 22 kg, but his spastic diplegia symptoms had not improved.", 'Patient concerns:': 'A Chinese boy initially presented with severe stunting and partial growth hormone deficiency (PGHD) at 3 years old and was initially treated with growth hormone replacement therapy. Seven years later (at 10 years old), he presented with spastic diplegia, cognitive function lesions, epilepsy, and peripheral neuropathy.'}
IEM-Treatment
IEM_Treatment
[ "Following the diagnosis , we recommended that the patient begins a low - protein diet ( 0.8 g / kg / day ) to decrease his blood arginine levels , as well as start taking citrulline ( 150–200 mg / kg / day ) ." ]
test-1567
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Vitals-and-Hematology
Vitals_Hema
[ "The patient 's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight - for - height Z - score ( −0.3 ) was in the 15th–50th percentile of the World Health Organization ( WHO ) growth standards", "Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight - for - height Z - score ( −1.6 ) decreased to the 3rd–15th percentile of the WHO growth standards" ]
test-1568
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Gastrointestinal-System
GI
[ "swallowing difficulty", "symptoms of aspiration while swallowing and prolonged feeding time", "irregularly ingested a soft and blended diet", "She had difficulty in eating and she showed signs of aspiration ( e.g., coughing, gagging )" ]
test-1569
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Patient-History
History
[ "A 22 - month - old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control" ]
test-1570
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Neurology
Neuro
[ "chief complaints of spasticity and swallowing difficulty as well as difficulty with head control.", "She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time.", "At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale ( MAS ). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross - motor domain, by 3 months in language and fine motor domain, and by 2 months in personal - social interaction domain in the Denver Developmental Screening Test II.", "She had difficulty in eating and she showed signs of aspiration ( e.g., coughing, gagging ). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas" ]
test-1571
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Laboratory-and-Imaging
Lab_Image
[ "Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement", "The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A ( % ) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol / hr / mg ( reference range, 620–1,000 nmol / hr / mg ) and 74.3 % ( reference range, 55%–72 % ), respectively", "videofluoroscopic swallowing study ( VFSS ), and was given barium - containing free water, yogurt ( thick liquid ), and rice porridge ( soft ). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time ( 1.3 seconds ) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing" ]
test-1572
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Cardiovascular-System
CVS
[]
test-1573
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Endocrinology
ENDO
[]
test-1574
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Genitourinary-System
GU
[]
test-1575
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Respiratory-System
RESP
[ "symptoms of aspiration while swallowing", "signs of aspiration ( e.g., coughing, gagging )" ]
test-1576
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Musculoskeletal-System
MSK
[ "genu recurvatum" ]
test-1577
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Eyes-Ears-Nose-Throat
EENT
[ "the patient did not show cherry red spot on fundoscopy" ]
test-1578
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Dermatology
DERM
[]
test-1579
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Pregnancy
Pregnancy
[ "born at full term through normal vaginal delivery" ]
test-1580
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Lymphatic-System
LYMPH
[]
test-1581
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Age-at-Presentation
Age (at case presentation)
[ "22 - month - old" ]
test-1582
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Age-of-Onset
Age (of onset)
[]
test-1583
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
Confirmed-Diagnosis-IEM
Confirmed_Diagnosis(IEM)
[ "Sandhoff disease", "the patient was diagnosed with Sandhoff disease ." ]
test-1584
5698679
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
{'CASE REPORT': "A 22-month-old girl diagnosed with Sandhoff disease was referred to our hospital with chief complaints of spasticity and swallowing difficulty as well as difficulty with head control. She was born at full term through normal vaginal delivery. She was able to stand while holding on to something at 8 months of age, which suggested normal development. However, developmental arrest occurred at 13 months of age. Spasticity and genu recurvatum were observed while standing and holding on to something. The patient showed a progressive motor regression pattern, and at 18 months of age, she could barely maintain a propped sitting position. At 20 months of age, she showed symptoms of aspiration while swallowing and prolonged feeding time. Brain magnetic resonance imaging scans obtained at 15 months of age did not show delayed myelination or abnormal enhancement ( Fig. 1 ). Developmental regression was similar to that in GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The patient underwent hexosaminidase assay in white blood cells, which showed increased hexosaminidase A (%) and decreased total hexosaminidase. The total hexosaminidase A & B and hexosaminidase A levels were 321.2 nmol/hr/mg (reference range, 620–1,000 nmol/hr/mg) and 74.3% (reference range, 55%–72%), respectively. Based on these findings, the patient was diagnosed with Sandhoff disease. However, the patient did not show cherry red spot on fundoscopy. At the time of admission, physical examination revealed grade 3 and 2 muscle strength in the upper and lower extremities, respectively. Neurological examination showed sustained ankle clonus and increased deep tendon reflexes in all extremities as well as hypertonicity and decerebrate posture manifested by an exaggerated extensor posture of all extremities, while crying and/or when she was irritable ( Fig. 2 ). In terms of spasticity, her four extremities scored grade 2 based on the modified Ashworth scale (MAS). Overall, the patient had developmental regression. Especially, she showed a delay in development by 1 month in gross-motor domain, by 3 months in language and fine motor domain, and by 2 months in personal-social interaction domain in the Denver Developmental Screening Test II. Dietary assessment revealed that the patient irregularly ingested a soft and blended diet of 70–130 kcal, five times a day, and the mean eating duration was approximately 1 hour. She had difficulty in eating and she showed signs of aspiration (e.g., coughing, gagging). Moreover, she had a poor feeding posture with inadequate trunk and head support. Oral motor function assessment showed that the patient had increased tone and decreased movement of the tongue and hypersensitivity in the oral and perioral areas. The patient underwent videofluoroscopic swallowing study (VFSS), and was given barium-containing free water, yogurt (thick liquid), and rice porridge (soft). In the oral phase, motor control, including lip sealing, tongue control, and mastication, was inadequate with delayed oral transit time (1.3 seconds) and premature bolus loss. In the pharyngeal phase, a delayed swallow reflex was observed without aspiration or penetration during or after swallowing ( Fig. 3 ). The patient's height and body weight were 85.0 cm and 11.6 kg, respectively. Her weight-for-height Z-score (−0.3) was in the 15th–50th percentile of the World Health Organization (WHO) growth standards. Nutritional evaluation showed that the patient needed a regular daily intake of 989 kcal. However, she could only consume 700 kcal/day, which was less than the required caloric intake. The patient was given supplementary balanced nutrition twice a day. The patient received an intensive dysphagia rehabilitation program for 30 minutes a day, five times a week, for 6 weeks, and it comprised both direct and indirect interventions, such as oromotor facilitation, thermo-tactile stimulation, and lip strengthening, and compensatory techniques, such as postural modification and alteration of the feeding utensil to facilitate swallowing. She was given anti-spasticity drugs for managing spasticity and achieving a stable posture during feeding. After the treatment, she was fed in a semi-reclined position, which reduced the signs of aspiration and the amount of food or liquid leakage from the mouth. However, her nutritional status showed no improvement, with her daily calorie intake decreasing to 600 kcal/day after 4 weeks of admission. At 6 weeks postadmission, her total intake further decreased to 392 kcal/day. Her body weight continuously declined from 11.6 kg to 10.5 kg, and her weight-for-height Z-score (−1.6) decreased to the 3rd–15th percentile of the WHO growth standards, which was a sharp decrease of one major percentile line in a short period. Gastrostomy was therefore recommended, but her parents refused the procedure. She was then referred to another hospital for treatment of deconditioning due to dehydration, and she received gastrostomy."}
IEM-Treatment
IEM_Treatment
[]
test-1585
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Vitals-and-Hematology
Vitals_Hema
[ "vital signs on admission showed uncontrolled hypertension ( BP = 148/118 mm Hg ) and tachycardia ( HR = 120 bpm )." ]
test-1586
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Gastrointestinal-System
GI
[ "history of recurrent abdominal pain", "nausea, vomiting, worsening of abdominal pain, and constipation", "unable to eat", "abdominal pain persisted", "abdominal pain resolved in less than 24 h" ]
test-1587
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Patient-History
History
[ "This is a 20 - year - old female with a 6 - month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days" ]
test-1588
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Neurology
Neuro
[ "two generalized tonic – clonic seizures", "she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region", "She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid ( CSF ) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus. The brain MRI showed PRES", "cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording", "neurological exam was normal", "follow - up brain MRI showed remarkable improvement" ]
test-1589
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Laboratory-and-Imaging
Lab_Image
[ "Head CT was normal, cerebral spinal fluid ( CSF ) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus. The brain MRI showed PRES", "Urine, then serum and fecal porphyrins were requested. The results were as follows : urine PBG level was 177.4 μmol / L ( reference : 0 to 0.88 μmol / L ), serum porphyrin level was 58 nmol / L ( reference : 0 to 15 nmol / L ), fecal coproporphyrin was 18 nmol / g ( reference : 0 to 45 nmol / g ), and fecal protoporphyrin was 10 nmol / g ( reference : 0–100 nmol / g", "highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels", "follow - up brain MRI showed remarkable improvement." ]
test-1590
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Cardiovascular-System
CVS
[]
test-1591
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Endocrinology
ENDO
[]
test-1592
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Genitourinary-System
GU
[ "acute pyelonephritis," ]
test-1593
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Respiratory-System
RESP
[]
test-1594
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Musculoskeletal-System
MSK
[]
test-1595
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Eyes-Ears-Nose-Throat
EENT
[]
test-1596
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Dermatology
DERM
[]
test-1597
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Pregnancy
Pregnancy
[]
test-1598
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Lymphatic-System
LYMPH
[]
test-1599
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Age-at-Presentation
Age (at case presentation)
[ "a 20 - year - old" ]
test-1600
5021915
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
{'Case presentation': "This is a 20-year-old female with a 6-month history of recurrent abdominal pain. She underwent cholecystectomy 3 months prior to admission but had no relief of her symptoms. She was admitted with acute pyelonephritis, nausea, vomiting, worsening of abdominal pain, and constipation for three days. Her vital signs on admission showed uncontrolled hypertension (BP = 148/118 mm Hg) and tachycardia (HR = 120 bpm). She was unable to eat during the last few days. On the second day of her admission, she had two generalized tonic–clonic seizures. She received lorazepam and levetiracetam, and her clinical seizures resolved; however, she continued to be lethargic, and a stat EEG was requested, that showed LPDs + F over the right temporoparietal region. The patient was transferred to the ICU for blood pressure control and was started on treatment with acyclovir, antihypertensive medications, and antibiotics. She became profoundly lethargic and assumed a fetal position in bed. On neurological examination, there were left lower extremity clonus and a left Babinski sign. Head CT was normal, cerebral spinal fluid (CSF) was unremarkable, and comprehensive CSF profiles were negative, including PCR for Herpes Simplex virus, Varicella Zoster virus, Lyme disease, and Cryptococcus . The brain MRI showed PRES ( Fig. 1 ). Because the stat EEG showed LPDs + F, a highly epileptogenic pattern, cEEG was initiated to rule out the possibility of recurrent electrographic seizures or nonconvulsive status epilepticus ( Fig. 2 ). There was one electrographic seizure arising from the right temporoparietal region lasting less than 1 min seen during the first 12 h of recording but none afterwards during the next two days of recording. Therefore, clinical and electrographic seizures completely subsided with treatment. Acyclovir was discontinued, and high dose steroids were started, considering the possibility of immunological and inflammatory etiologies. The patient showed clinical improvement, but her abdominal pain persisted, and the diagnosis of an acute porphyria was suspected. Urine, then serum and fecal porphyrins were requested. The results were as follows: urine PBG level was 177.4 μmol/L (reference: 0 to 0.88 μmol/L), serum porphyrin level was 58 nmol/L (reference: 0 to 15 nmol/L), fecal coproporphyrin was 18 nmol/g (reference: 0 to 45 nmol/g), and fecal protoporphyrin was 10 nmol/g (reference: 0–100 nmol/g). Taken together, the highly increased urine PBG levels, slightly increased plasma porphyrin levels, and normal fecal porphyrin levels, were suggestive of AIP. The patient's abdominal pain resolved in less than 24 h with carbohydrate (glucose) loading and highly caloric diet. Therefore, intravenous hemin was not necessary at that time. She was discharged home in stable conditions and offered to follow up at a porphyria center of her choice for further confirmatory genetic testing, management, and counseling. The patient was seen in our epilepsy outpatient clinic one month later. She was not taking any medications. She was asymptomatic, and her neurological exam was normal. A follow-up brain MRI showed remarkable improvement."}
Age-of-Onset
Age (of onset)
[]