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Editor’s key points
fatigue
RECRUITMENT
Anxiolysis for children undergoing short day-case surgical procedures is an important intervention.The findings of this randomised trial suggest melatonin is less effective than midazolam for preoperative anxiolysis in children.However, the trial was terminated early because of recruitment fatigue, which makes the fin...
PMC10797512
Methods
PMC10797512
Study design and participants
learning disabilities, throat, anxiety
BLIND
This multicentre, parallel-group, double blind, individual participant-randomised trial took place across 20 UK NHS Hospital Trusts. Delegated research staff identified, assented, and randomised paediatric participants identified as anxious on the day of surgery. Trial information was given via age-specific patient inf...
PMC10797512
Randomisation and blinding
’s taste reaction, head and neck, gastroenterology and MRI
Participants were randomly assigned in a 1:1 ratio to either control (midazolam) or treatment (melatonin) arms. Randomisation was computer-generated and completed using minimisation based on centre, surgical speciality (head and neck, gastroenterology and MRI, and other) and sex (male/female). All hospitals involved in...
PMC10797512
Data collection
anxiety
A series of anxiety and recovery measures, for both child participants and their caregivers, were administered on the day of surgery (pre- and post-surgery) and 14 days post-discharge.
PMC10797512
Interventions
Participants were allocated to receive either melatonin or midazolam 0.5 mg kg
PMC10797512
Primary outcome measure—modified Yale Preoperative Anxiety Scale-Short Form
Anxiety, anxiety
The primary endpoint was modified Yale Preoperative Anxiety Scale-Short Form (mYPAS-SF) score (adjusted for baseline) measured over the three consecutive, standard preoperative time points recommended for the scale: start of transfer to theatre, entry into anaesthetic room, and administration of anaesthesia. The mYPAS-...
PMC10797512
Secondary outcome measures
anxiety
SECONDARY
A selection of secondary outcome measures were recorded to further assess participant and caregiver preoperative anxiety and postoperative child recovery, including; anaesthetic failure, Cooperation Score, FPS-R observer and participant reported, caregiver STAI questionnaire, VSRS, and PAED index. At 2 weeks post-surge...
PMC10797512
Sample size
The sample size, based on mYPAS-SF over all three time points whilst adjusting for baseline (assumed correlation 0.5),
PMC10797512
Statistical analysis
All of the statistical analysis was performed according to the MAGIC Statistical Analysis Plan
PMC10797512
Approvals
The full MAGIC trial protocol is available via the University of Sheffield data repository, ORDA.
PMC10797512
Results
PMC10797512
Early closure
RECRUITMENT
The MAGIC trial opened to recruitment in July 2019. In early 2020, the COVID-19 pandemic severely disrupted the trial, which was halted and then reopened to recruitment in October 2020, closed again in February 2021, and re-opened again in June 2022. However, the ongoing pandemic led to challenges in drug supply, paedi...
PMC10797512
Recruitment, randomisation, and withdrawal
throat, Anxiety
Between July 30, 2019 and November 9, 2022, 110 participants were randomised across 17 centres; 55 to receive melatonin and 55 to receive midazolam (CONSORT flow diagram. mYPAS-SF, modified Yale Preoperative Anxiety Scale-Short Form; PI, principal investigator; RN, research nurse.Baseline characteristics of randomised ...
PMC10797512
Primary outcome measure (mYPAS-SF)
The primary outcome measure, mYPAS-SF, was completed at baseline and at least one follow-up time point for 94 participants (86%), with all data included in the ITT analysis. Six participants (6%) were excluded from the PP analysis. The adjusted mean difference in mYPAS-SF scores for melatonin Adjusted mean difference i...
PMC10797512
Secondary outcome measures
SECONDARY
Full results from all of the secondary outcomes can be found in Post-surgery, PAED index decreased over time from a mean score of 11.6 (VSRS scores increased over time from a mean score of 13.7 (
PMC10797512
Adverse events
ADVERSE EVENTS, ADVERSE EVENT
Of the participants receiving the IMP (99/110, 90%), 22 (22%) had at least one AE (Number of adverse events and proportion of participants with at least one adverse event.
PMC10797512
Discussion
delirium, Delirium
SECONDARY, RECRUITMENT
Here we report the first multicentre, randomised trial of melatonin The magnitude of difference in adjusted mYPAS-SF observed in the midazolam group compared with melatonin was a consistent finding throughout all three time points (transfer, entry into anaesthetic room, and anaesthetic induction). Consequently, both IT...
PMC10797512
Future studies
anxiety
The trial showed that using melatonin as a premedication was less effective at reducing preoperative anxiety than midazolam, further adding to the knowledge base within the perioperative anaesthetic field of effective anxiolytic paediatric premedications. However, given the findings of the MAGIC trial, there still rema...
PMC10797512
Conclusions
anxiety
The trial did not reach the required sample size and therefore is prone to bias. Within the population studied, melatonin is less effective than midazolam at reducing preoperative anxiety in children and young people before general anaesthesia. The current standard of care, midazolam, was better by a clinically meaning...
PMC10797512
Authors' contributions
RS
CD (chief investigator), RB (co-investigator), MCH (trial manager), EH (statistician), DP (CTRU lead), NT (statistician) and MW (co-investigator) together produced the first draft of the manuscript. All members of the authorship group, provided in Conceived of or designed the work: RB, DP, CD, NT, MW, ZM, JC, SoA, SA, ...
PMC10797512
Declaration of interest
The authors declare that they have no conflicts of interest.
PMC10797512
Funding
The MAGIC trial was funded by the NIHR Health Technology Assessment programme (NIHR HTA 16/80/08). Further information available at:
PMC10797512
References
PMC10797512
Supplementary data
The following are the Supplementary data to this article:
PMC10797512
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PMC10797512
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PMC10797512
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PMC10797512
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PMC10797512
Acknowledgements
SCHILDER
We gratefully acknowledge the hard work, support, and advice from the following: the MAGIC study teams, research nurses; research support staff and clinical teams within the participating NHS trusts for participant screening, delivering the intervention, data collection, and patient follow up. We also offer thanks to A...
PMC10797512
Subject terms
infection, cardiac dysfunction
INFECTION, SARS-COV-2 INFECTION, CARDIOVASCULAR DISORDERS
Post-COVID-19 condition refers to a range of persisting physical, neurocognitive, and neuropsychological symptoms following SARS-CoV-2 infection. Recent evidence revealed that post-COVID-19 syndrome patients may suffer from cardiac dysfunction and are at increased risk for a broad range of cardiovascular disorders. Thi...
PMC10257166
Introduction
cardiac injury, COVID-19 syndrome, cardiac abnormalities, myocardial inflammation
CORONAVIRUS, MYOCARDIAL INFLAMMATION, SEVERE ACUTE RESPIRATORY SYNDROME
The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic has resulted in more than 541 million infected cases, as of June 2022. Even though most infected patients recover, around 10–30% remain with persistent symptoms that can significantly affect their quality of lifeThe long-term impact of COVID-19 a...
PMC10257166
Methods
PMC10257166
Patients
cognitive decline, traumatic brain injury
ASYMPTOMATIC SARS-COV-2 INFECTION, BRAIN PATHOLOGY
Included patients were over 18 years old with a reported post-COVID-19 cognitive symptoms that affected their quality of life and persisted for more than three months after an RT-PCR confirmed mild-moderate symptomatic SARS-CoV-2 infection. Patients were excluded if they had a history of pathological cognitive decline,...
PMC10257166
Design
SECONDARY
The current study is part of phase II exploratory clinical trial. A prospective randomized, double-blind, sham-controlled study was conducted from December 14, 2020, to December 27, 2021, at Shamir Medical Center (SMC), Israel. After signing informed consent, patients were randomized to either HBOT or sham-control grou...
PMC10257166
Intervention
Both HBOT and sham protocols were administrated in a multi-place Starmed-2700 chamber (HAUX, Germany). The protocol comprised of 40 daily sessions, five sessions per week within a two-month period. The HBOT protocol included breathing 100% oxygen by mask at 2ATA for 90 min with five-minute air breaks every 20 min. Comp...
PMC10257166
Primary and secondary outcomes
PMC10257166
Primary outcomes—Global Longitudinal Strain
Secondary outcomes: myocardial work index parameters: Global Work Index, Global Constructive Work. Global Wasted Work, Global Work Efficacy.
PMC10257166
Echocardiography examination
All echocardiography exams were performed using Vivid E95, (General Electric; Horten, Norway) with a standard transducer of 1.7–4 Hz. The frame rate during echocardiography examinations was greater than or equal to 40 frames per second. Comprehensive transthoracic echocardiography examinations were performed according ...
PMC10257166
Statistical analysis
Continuous data were expressed as means ± standard deviations (SD). Normality assumption was evaluated according to a Kolmogorov–Smirnov test. A paired t-test and a two-sample Student’s t-test were used to compare means as appropriate. A Welch's 
PMC10257166
Results
intercurrent illness, traumatic
WASTED, EVENT
Out of 91 patients eligible to participate in the study, 11 patients did not complete the baseline evaluation and one patient did not meet inclusion criteria. Thus, 79 patients were randomized to either HBOT or sham arms. Two patients from the sham group withdrew their consent during treatment, and two patients did not...
PMC10257166
Discussion
myocardial damage, fatigue, early-stage myocardial fibrosis, COVID-19 infection, myocardial dysfunction, systolic dysfunction, cardiac abnormalities
VIRUS, NEUROLOGICAL DISEASE, LEFT VENTRICULAR DYSFUNCTION, POST-ACUTE COVID-19 SYNDROME, COVID-19 INFECTION, CARDIAC COMPLICATIONS, MYOCARDIAL DYSFUNCTION, SYSTOLIC DYSFUNCTION
The current randomized controlled trial demonstrates a subtle systolic dysfunction of GLS lower than − 20%, in about half (48.3%) of the post-COVID-19 syndrome patients, which was significantly improved by HBOT. This recovery in GLS by HBOT exceeded the natural recovery rate observed in the sham group.Previous studies ...
PMC10257166
Author contributions
M.L.—writing of the manuscript, data analysis; S.F.—a critical review, intellectual input; V.T.—data analysis, intellectual input; critical review; A.H.—a critical review, intellectual input; S.Z.-Y.—a critical review; S.E.—sudy design, intellectual input, critical review, final approval. All authors reviewed the manus...
PMC10257166
Data availability
The datasets analyzed during the current study available from the corresponding author on reasonable request.
PMC10257166
Competing interests
The authors declare no competing interests.
PMC10257166
References
PMC10257166
Background
COLORECTAL CANCER, SOLID TUMORS
NEO201 is a humanized IgG1 monoclonal antibody (mAb) generated against tumor-associated antigens from patients with colorectal cancer. NEO-201 binds to core 1 or extended core 1 O-glycans expressed by its target cells. Here, we present outcomes from a phase I trial of NEO-201 in patients with advanced solid tumors that...
PMC10053355
Methods
toxicity
DISEASE PROGRESSION, SECONDARY, DISEASE
This was a single site, open label 3 + 3 dose escalation clinical trial. NEO-201 was administered intravenously every two weeks in a 28-day cycle at dose level (DL) 1 (1 mg/kg), DL 1.5 (1.5 mg/kg) and DL 2 (2 mg/kg) until dose limiting toxicity (DLT), disease progression, or patient withdrawal. Disease evaluations were...
PMC10053355
Results
toxicities, SD, neutropenia, febrile neutropenia
DISEASE PROGRESSION, COLORECTAL CANCER, NEUTROPENIA, BREAST CANCERS, FEBRILE NEUTROPENIA, DISEASE
Seventeen patients (11 colorectal, 4 pancreatic and 2 breast cancers) were enrolled; 2 patients withdrew after the first dose and were not evaluable for DLT. Twelve of the 15 patients evaluable for safety discontinued due to disease progression and 3 patients discontinued due to DLT (grade 4 febrile neutropenia [1 pati...
PMC10053355
Conclusions
treatment-resistant solid tumors, neutropenia
ADVERSE EVENT, NEUTROPENIA
NEO-201 was safe and well tolerated at the MTD of 1.5 mg/kg, with neutropenia being the most common adverse event. Furthermore, a reduction in the percentage of regulatory T cells following NEO-201 treatment supports our ongoing phase II clinical trial evaluating the efficiency of the combination of NEO-201 with the im...
PMC10053355
Graphical Abstract
PMC10053355
Supplementary Information
The online version contains supplementary material available at 10.1186/s13046-023-02649-6.
PMC10053355
Keywords
PMC10053355
Background
colorectal cancer, tumor, neutropenia, toxicity, cancer, NEO-201, toxicities, tumors
TUMOR, COLORECTAL CANCER, NEUTROPENIA, CARCINOGENESIS, CANCER, DISEASE, SOLID TUMORS, ADVERSE EFFECT, TUMORS, CYTOTOXICITY
Recent efforts in cancer therapeutics have focused on the development of drugs that activate the immune system against cancer cells to achieve durable disease control. In recent decades, cancer immunotherapies have emerged as promising treatment options for many cancer patients and have increasingly been employed in sp...
PMC10053355
Methods
PMC10053355
Study design and patient selection
tumor, DLTs, bleeding, neutropenia, febrile neutropenia, toxicity, toxicities, thrombocytopenia, signet cell ovarian cancer, anemia, pancreatic cancer, Cancer
TUMOR, BLEEDING, FEBRILE NEUTROPENIA, ADENOCARCINOMA OF THE LUNG, CANCER, BREAST CANCER, THROMBOCYTOPENIA, SQUAMOUS CELL LUNG CANCER, NEUTROPENIC FEVER, COLON CANCER, ANEMIA, METASTATIC CANCER, PANCREATIC CANCER, CARCINOMATOSIS, DISEASE PROGRESSION, NEUTROPENIA, EVENT, DISEASE, SECONDARY, PLEURAL EFFUSIONS, INFUSION RE...
This is a Phase I first-in-human, single center, open label, dose escalation clinical trial (NCT03476681) using a standard 3 + 3 design. The primary objective was to determine the RP2D of NEO-201. The secondary objective was to assess the preliminary antitumor activity of NEO-201 and exploratory aims characterized the ...
PMC10053355
Safety assessments
toxicity, DLTs
ADVERSE EVENTS, ADVERSE EVENT, ADVERSE EVENT
All patients who received at least two doses of NEO-201 were evaluable for safety and toxicity unless they were removed from study therapy for DLT. Safety evaluations were conducted at every treatment cycle, including determination of adverse events, DLTs during the dose-escalation stage, clinical laboratory measuremen...
PMC10053355
Efficacy assessments
tumor
DISEASE PROGRESSION, DISEASE, PROGRESSION, TUMOR
Patients were considered evaluable for clinical response if they had measurable disease present at baseline, received at least one cycle of therapy, developed objective disease progression prior to the end of cycle 1, or had their disease re-evaluated by imaging at the conclusion of cycle 1. Radiologic assessment, incl...
PMC10053355
Pharmacokinetic (PK) analyses
cancer
BLOOD, CANCER
Blood samples for PK analysis were obtained at pre-dose prior to the first infusion dose on cycle 1 day 1 (C1D1), end-of-infusion (EOI), then at 1 h (hr) post EOI, 4 h post EOI, 24 h post EOI, 72 h post EOI, 7 days post EOI and 14 days post EOI. Spare sampling (pre/trough and end of infusion) was performed on cycle 1 d...
PMC10053355
Immunohistochemistry (IHC)
colon carcinoma, cancer, tumor, Cancer
COLON CARCINOMA, CANCER, TUMOR, CANCER
Archival tumor tissue samples were collected from all patients enrolling in the study, but expression of NEO-201 target antigen was not an eligibility criterion. A qualitative IHC staining system was used to identify the expression of the antigen recognized by NEO-201 in formalin-fixed, paraffin-embedded (FFPE) neoplas...
PMC10053355
Correlative assays
PMC10053355
Cytokines
toxicity, CRS
BLOOD, CLOT, CYTOKINE RELEASE SYNDROME
Blood samples (10 mL) were drawn for cytokine analysis in 10 mL red-top tubes to evaluate the toxicity risk of cytokine release syndrome (CRS). Blood samples were drawn prior to the first infusion dose on C1D1, 24 h post EOI, 72 h post EOI, 14 days post EOI and prior to C3D1. After drawing, blood was allowed to clot at...
PMC10053355
Soluble factors
BLOOD
Blood samples (10 mL) were drawn in 10 mL red-top tubes to evaluate the serum levels of soluble CEACAM-5, CEACAM-6 and MICA. Blood samples were drawn prior to the first infusion dose on C1D1, 72 h post EOI, 14 days post EOI and prior to C3D1. After drawing, blood was processed with the same procedure described for cyto...
PMC10053355
NK cells and regulatory T cells (Tregs) phenotype analysis
cancer
CANCER, COLD, APC
Analysis of the expression of cell-surface and intracellular proteins in peripheral blood mononuclear cells (PBMCs) from cancer patients was performed by flow cytometry to evaluate NK cells and Tregs phenotype. PBMCs from cancer patients were utilized under the appropriate NCI Institutional Review Board approval (proto...
PMC10053355
Statistical analysis
Student’s t-test, 1-way ANOVA, and 2-way ANOVA with Bonferroni post-test analysis were performed where indicated. The number of samples chosen for each comparison was determined based on past similar experiments or by performing pilot experiments to assess the expected magnitude of differences. The number of experiment...
PMC10053355
Results
PMC10053355
Study population and disposition
colorectal cancer, pancreatic cancer, breast cancer
COLORECTAL CANCER, PANCREATIC CANCER, BREAST CANCER
Between January 8, 2019 and December 8, 2020, a total of 17 patients received one or more doses of NEO-201. The demographics of patients enrolled are listed in Table Patient demographics and baseline characteristicsPrior to treatment with NEO-201, ten and six patients with colorectal cancer had received anti-VEGF and a...
PMC10053355
Toxicity
neutropenia, febrile neutropenia, necrotic tumor, toxicity, infection
NEUTROPENIA, FEBRILE NEUTROPENIA, NECROTIC TUMOR, ADVERSE EVENTS, INFECTION
Among the 17 patients enrolled, 4 patients received NEO-201 at DL 1 (1 mg/kg), 7 patients received NEO-201 at DL 2 (2 mg/kg) and 6 patients received NEO-201 at DL 1.5 (1.5 mg/kg) (Table Most common grade 3 (Gr3) and grade 4 (Gr4) adverse events (AEs) in all patientsSee Supplementary Table Due to the ability of NEO-201 ...
PMC10053355
Activity
colorectal cancer, SD
DISEASE, COLORECTAL CANCER
Thirteen patients were able to undergo assessment for disease response. The best response observed was SD (> 56 days) in 4 patients, all of whom had colorectal cancer (Fig. RECIST response and time on study All 4 patients with SD after at least 4 doses of NEO-201 elected to continue receiving therapy, range of 1–11 add...
PMC10053355
Pharmacokinetic analyses
All 17 patients were evaluable for first dose noncompartmental pharmacokinetic analysis. First dose NEO-201 serum concentration–time profiles were measured for all 17 patients across the three different dose levels (Fig. Clinical Pharmacokinetics of NEO-201 Relevant PK parameters during dense PK sampling were calculate...
PMC10053355
Time of administration of filgrastim affects NEO-201 PK
neutropenia
NEUTROPENIA
Ten of 17 patients (4 in DL 2 and 6 in DL 1.5) received filgrastim to shorten the duration of the neutropenia.In the DL 2 cohort, time of administration of filgrastim to mitigate neutropenia had an impact on NEO-201 concentration in the serum of some patients (Supplementary Fig. 
PMC10053355
Pharmacodynamic analyses
PMC10053355
Immunohistochemistry (IHC)
tumor, breast cancer
TUMOR, BREAST CANCER
Baseline tissue samples from all patients enrolled in the study were tested for NEO-201 expression by IHC. Fifteen of 17 patients had tissue evaluable for IHC, and of these 14/15 had more than 90% tissue stain positive for the NEO-201 antigen with 3 + intensity. One breast cancer patient had 3 + staining in about 20% o...
PMC10053355
Cytokine analysis
TNF-α
PANCREATIC CANCER
To evaluate modulation of cytokines by NEO-201, serum samples were collected at timepoints before and after treatment and analyzed by ELISA-based cytokine array. At 24 h from infusion, serum IL-10 and TNF-α levels were increased in all patients at all dose levels. IL-10 increased to median fourfold higher in all patien...
PMC10053355
Soluble MICA affects NK cell phenotype
cancer
CANCER, CYTOTOXICITY
The release of soluble factors from cancer cells constitutes an immune escape mechanism that systemically impairs efficacy of immunotherapy [Correlation between soluble factors and immune cells anti-cancer activity Since it has been demonstrated that tumor-derived soluble MICA can negatively impact NK cell cytotoxicity...
PMC10053355
NEO-201 binds to circulating Tregs
In a previous study, flow cytometry analysis of hematopoietic cells for NEO-201 binding revealed that approximately 4.6% of CD4One marker used to characterize a subset of highly immunosuppressive Tregs is CD15s [
PMC10053355
Discussion
NSCLC, tumor, colorectal cancer, TNF-α, neutropenia, toxicity, cancer, inflammation, Head and Neck Squamous Cell Carcinoma, SD, uterine carcinoma, PD, NRAS, HNSCC, tumors
TUMOR, METASTASIS, COLORECTAL CANCER, NEUTROPENIA, ONCOGENESIS, CANCER, INFLAMMATION, EVENT, SOLID TUMORS, ADVERSE EFFECT, NON-SMALL CELL LUNG CANCER, UTERINE CARCINOMA, OVARIAN CARCINOMA, CERVICAL CANCER, NSCLC, TUMORS, COMPLICATIONS
One of the mechanisms involved in cancer development, progression and metastasis is the disruption of post-translational modifications of proteins and lipids, such as glycosylation. One glycosylation pattern altered in cancer cells is the O-glycosylation. During oncogenesis, truncated O-glycans can be expressed. This o...
PMC10053355
Conclusions
NSCLC, pembrolizumab, HNSCC, uterine cancer, chemo-resistant solid tumors, SD, malignancies, tumors
SOLID TUMORS, MALIGNANCIES, CERVICAL CANCER, NSCLC, TUMOR GROWTH, TUMORS
This first-in-human study of NEO-201 in solid tumors demonstrated that NEO-201 was well-tolerated and the RP2D was established at 1.5 mg/kg. Exploratory studies in serum suggested a correlation between maintenance of SD and lower baseline levels of soluble serum MICA, while serum levels of CEACAM-5 and CEACAM-6 were no...
PMC10053355
Acknowledgements
Authors want to thank Seth Steinberg, head of Biostatistics and Data Management Section at NCI, for his biostatistical help.
PMC10053355
Authors’ contributions
MPM
RECRUITMENT
Conceptualization and design of clinical trial: CBC, MPM, MF, SAM, KYT, CMA; Recruitment of patients: CBC, MPM, NH, AMC, CMA; methodology, data interpretation and validation (pharmacokinetics): CP, WDF,TY; methodology, data interpretation and validation (IHC): MM, PF; methodology, data interpretation, and validation (c...
PMC10053355
Funding
The study has been funded by Precision Biologics, Inc. and by NCI.
PMC10053355
Declarations
PMC10053355
Ethics approval and consent to participate
The study was approved by NCI, NIH, Institutional Review Board (protocol code NCT03476681, first approved 03/26/2018; latest update 01/08/2020) and all participants signed a written informed consent.
PMC10053355
Availability of data and materials
All data generated or analyzed during this study are included in this published article [and its supplementary information files].Data not shown in the manuscript are available from the corresponding author on reasonable request.
PMC10053355
Consent for publication
All patients gave written informed consent.All authors have read and approved of its submission to this journal.
PMC10053355
Competing interests
MF, SAM, AZ, KYT and PMA are employees of Precision Biologics, Inc. PMA has an ownership interest in Precision Biologics, Inc. PMA had no role in the design of the study, in the collection, analyses or interpretation of the data, in the writing of the manuscript or in the decision to publish the results. All other auth...
PMC10053355
References
PMC10053355
Key Points
PMC10308298
Question
psychosis
What are the optimal type, timing, and sequence of interventions for individuals at ultrahigh risk of psychosis?
PMC10308298
Findings
REMISSION, RELAPSE
In this sequential multiple assignment randomized trial including 342 individuals, a specialized psychological intervention (cognitive-behavioral case management [CBCM]) and a psychopharmacological intervention (CBCM and antidepressant medication) were not more efficacious than control conditions in improving remission...
PMC10308298
Meaning
psychosis
The findings of this study show that addition of sequentially more specialized psychosocial and antidepressant treatment for individuals who did not remit did not lead to superior outcomes, underscoring the need for further adaptive trials, treatment innovation, and an extended duration of care for relapse prevention.T...
PMC10308298
Importance
psychosis
Clinical trials have not established the optimal type, sequence, and duration of interventions for people at ultrahigh risk of psychosis.
PMC10308298
Objective
psychosis
To determine the effectiveness of a sequential and adaptive intervention strategy for individuals at ultrahigh risk of psychosis.
PMC10308298
Design, Setting, and Participants
Psychosis, psychosis
The Staged Treatment in Early Psychosis (STEP) sequential multiple assignment randomized trial took place within the clinical program at Orygen, Melbourne, Australia. Individuals aged 12 to 25 years who were seeking treatment and met criteria for ultrahigh risk of psychosis according to the Comprehensive Assessment of ...
PMC10308298
Interventions
ω-3 fatty acids
Step 1: 6 weeks of support and problem solving (SPS); step 2: 20 weeks of cognitive-behavioral case management (CBCM) vs SPS; and step 3: 26 weeks of CBCM with fluoxetine vs CBCM with placebo with an embedded fast-fail option of ω-3 fatty acids or low-dose antipsychotic medication. Individuals who did not remit progres...
PMC10308298
Main Outcomes and Measures
Depression, psychosis
REMISSION
Global Functioning: Social and Role scales (primary outcome), Brief Psychiatric Rating Scale, Scale for the Assessment of Negative Symptoms, Montgomery-Åsberg Depression Rating Scale, quality of life, transition to psychosis, and remission and relapse rates.
PMC10308298
Results
psychosis
REMISSION, RELAPSE
The sample comprised 342 participants (198 female; mean [SD] age, 17.7 [3.1] years). Remission rates, reflecting sustained symptomatic and functional improvement, were 8.5%, 10.3%, and 11.4% at steps 1, 2, and 3, respectively. A total of 27.2% met remission criteria at any step. Relapse rates among those who remitted d...
PMC10308298
Conclusions and Relevance
psychosis
REMISSION
In this sequential multiple assignment randomized trial, transition rates to psychosis were moderate, and remission rates were lower than expected, partly reflecting the ambitious criteria set and challenges with real-world treatment fidelity and adherence. While all groups showed mild to moderate functional and sympto...
PMC10308298