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patient_id
string
age_at_dx_yrs
float64
sex
string
race_ethnicity
string
geographic_region
string
socioeconomic_status
string
hemophilia_type
string
severity_class
string
factor_activity_pct
float64
mutation_type
string
inhibitor_high_risk_mutation
int64
prophylaxis_flag
int64
prophylaxis_type
string
factor_product_type
string
factor_dose_iu_kg
float64
factor_frequency
string
factor_trough_iu_dl
float64
factor_halflife_hr
float64
factor_adherence_pct
float64
factor_annual_iu
int64
factor_cost_usd_yr
int64
home_infusion_flag
int64
port_access_flag
int64
prophylaxis_start_age_months
float64
emicizumab_flag
int64
emicizumab_regimen
string
emicizumab_abr_reduction
float64
fitusiran_flag
int64
marstacimab_flag
int64
concizumab_flag
int64
inhibitor_flag
int64
inhibitor_titer_bu_ml
float64
inhibitor_class
string
inhibitor_onset_exposure_days
int64
inhibitor_high_risk_mutation_flag
int64
iti_flag
int64
iti_protocol
string
iti_success_flag
int64
iti_duration_months
int64
bypassing_agent
string
inhibitor_thrombosis_flag
int64
abr_annual
int64
joint_bleed_rate_annual
int64
target_joint_flag
int64
target_joint_count
int64
cns_bleed_flag
int64
gi_bleed_flag
int64
psoas_hematoma_flag
int64
bleed_hospitalization_flag
int64
isth_bat_score
int64
hjhs_score_annual
float64
wfh_bleeding_score
float64
synovitis_grade
string
pettersson_score
float64
mri_joint_score
float64
cartilage_loss_flag
int64
osteophyte_flag
int64
joint_space_loss_pct
float64
hemosiderin_flag
int64
subchondral_cyst_flag
int64
ankle_rom_deg
float64
knee_rom_deg
float64
elbow_rom_deg
float64
joint_replacement_flag
int64
joint_replacement_age_yrs
float64
gait_impairment_flag
int64
aptt_seconds
float64
vwf_antigen_iu_dl
float64
factor_recovery_iu_dl_per_iu_kg
float64
peak_thrombin_nm
float64
etp_nm_min
float64
d_dimer_ug_ml
float64
crp_mg_l
float64
il6_pg_ml
float64
hiv_positive
int64
hcv_positive
int64
hbsag_positive
int64
gene_therapy_flag
int64
gt_product
float64
gt_vector_dose_vg_kg
float64
gt_factor_expr_yr1_pct
float64
gt_factor_expr_yr2_pct
float64
gt_factor_expr_yr5_pct
float64
gt_abr_reduction
float64
gt_zero_bleed_flag
int64
gt_liver_enzyme_flag
int64
gt_immunosuppression_flag
int64
gt_nab_flag
int64
gt_expression_loss_flag
int64
gt_response
float64
mortality_flag
int64
mortality_cause
string
hospitalization_flag
int64
hospitalization_days_yr
int64
qol_sf36_pcs
float64
promis_pain_interference
float64
haem_a_qol
float64
phq9
int64
gad7
int64
work_days_missed_yr
int64
employment_status
string
catastrophic_bleeds_lifetime
int64
HC003-0000001
4.81
Male
White_non_Hispanic
Europe
Low
B
Severe
0.1
Other
0
1
Tertiary
Plasma_derived
37.2
Every_other_day
15.2
9.5
0.554
146,560
64,664
1
0
17
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
5
4
0
0
0
0
0
0
3
0.5
15.2
null
7.9
1
0
0
12.8
0
0
30.6
119.2
145
0
null
0
66.4
140.6
3.08
130.6
856.1
0.472
5.41
9.84
0
1
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
0
0
19.7
59.6
77.5
8
2
2
Employed_part
1
HC003-0000002
4.67
Male
Black_non_Hispanic
US
Low
A
Severe
0.1
Large_deletion
1
0
None_on_demand
Recombinant_EHL
21.3
Every_other_day
15.7
21.4
0.291
180,855
103,803
1
0
15.5
1
Monthly
0.929
0
0
0
0
0
null
0
1
0
null
0
0
null
0
2
2
0
0
0
0
1
0
15
40.9
11.9
Mild
46.5
12.5
0
0
46.8
0
1
34.3
140
145
1
38.4
1
102.8
56.8
2.57
146.6
774.9
0.398
15.73
2.89
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
Surgical_complication
0
0
30.3
46.6
73.6
9
13
9
Employed_full
2
HC003-0000003
4.77
Male
White_non_Hispanic
Latin_America
Middle
A
Mild
29.63
Missense
0
0
None_on_demand
Recombinant_EHL
36.6
3x_week
20.2
21.5
0.671
860,772
113,602
0
0
10.1
1
Biweekly
0.874
0
0
0
0
0
null
0
0
0
null
0
0
null
0
3
2
0
0
0
0
0
1
7
9.7
9.9
null
20.3
2
0
0
68.4
1
0
42.6
102.3
122.5
1
36.7
0
31.7
115.1
2.78
172.6
649.4
0.305
7.82
1.89
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
27.9
51.1
72.2
12
4
1
Employed_full
0
HC003-0000004
0.56
Male
Hispanic
US
Low
A
Severe
0.1
Large_deletion
1
1
Tertiary
Recombinant_EHL
42.8
Every_other_day
11.4
24
0.761
177,138
142,723
1
0
1
0
null
0
0
1
0
1
2.4
Low_titer
17
1
0
null
0
0
Emicizumab_bypass
0
6
4
0
0
0
0
0
1
26
0
21.8
Mild
31.2
7.2
1
0
41.8
0
0
30.8
140
145
0
null
0
76
32
2
99.1
992.1
0.227
5.74
1.8
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
HIV_AIDS
1
12
39.5
61.2
36.1
15
9
14
Employed_full
1
HC003-0000005
0.17
Male
Hispanic
US
Middle
A
Mild
39.66
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
25.9
Weekly_EHL
1
14.5
0.704
65,293
419,347
1
0
31.9
1
Biweekly
0.974
0
0
1
0
0
null
0
1
0
null
0
0
null
0
1
1
0
0
0
0
0
0
5
41.9
15.6
Moderate
37.7
1.8
0
0
19.4
0
0
45.2
110.8
124.9
0
null
1
61.9
92.9
2.52
86.2
643.7
0.423
3.49
48.17
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
22
56.3
63.6
6
8
16
Employed_full
2
HC003-0000006
2.91
Male
White_non_Hispanic
Europe
Low
A
Mild
13.49
Intron22_inversion
1
0
None_on_demand
Plasma_derived
28.3
Weekly_EHL
12.3
8.2
0.431
497,873
157,405
0
0
22
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
6
4
0
0
0
0
0
0
12
6.3
13
Moderate
63.6
1
0
1
10.1
0
0
37.7
102.2
115.2
0
null
0
60.1
113.4
1.75
126.2
904.5
0.322
2.34
3.51
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
CNS_bleed
0
0
34.8
49
74
1
3
5
Employed_full
1
HC003-0000007
2.82
Male
Other
Latin_America
Middle
A
Mild
14.6
Small_indel
0
0
None_on_demand
Recombinant_EHL
23.8
Biweekly_EHL
13.2
17.4
0.54
660,202
165,415
0
0
1
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
29
22
0
0
0
0
0
0
32
0
13.3
Moderate
27.3
14.6
1
0
43.8
0
0
49.9
88.5
119.9
0
null
0
31.4
120
2.47
60.8
869.8
1.659
3.58
7.84
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
61.2
69
80.8
11
6
2
Employed_part
4
HC003-0000008
6.25
Male
White_non_Hispanic
Asia_Pacific
Middle
A
Severe
0.1
Small_indel
0
0
None_on_demand
Recombinant_SHL
25.2
Weekly_EHL
5.8
8
0.789
726,163
232,031
1
0
23.7
0
null
0
0
1
0
0
0
null
0
0
0
null
0
0
null
0
36
27
0
0
0
1
0
1
14
1.1
18.8
null
0
17.6
0
1
17
0
1
42.8
140
127.7
0
null
0
26.4
97
1.93
56.4
857.1
0.698
9.62
11.17
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
3
39.2
47.5
59.1
13
2
3
Disability
1
HC003-0000009
0.16
Male
White_non_Hispanic
US
Middle
B
Mild
33.47
Missense
0
0
None_on_demand
Recombinant_SHL
41.7
Every_other_day
12.9
8
0.648
500,323
159,746
0
0
12.6
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
26
20
0
0
0
1
0
0
9
16.7
6.5
Mild
39.4
15.6
0
1
20.2
0
0
63
137.8
110.9
0
null
0
72.5
93.2
4
80.3
707.8
0.176
6.49
7.25
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
40.5
26.1
80.2
13
12
2
Disability
0
HC003-0000010
2.09
Male
Hispanic
US
Middle
A
Mild
10.61
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
20
Every_other_day
9.8
17.4
0.631
551,784
219,713
0
0
13
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
13
10
0
0
0
0
0
0
14
15.5
5
Mild
25.4
0
1
0
50.8
0
1
43.5
105.4
115.7
0
null
1
37.1
115.9
2.7
93.6
671
0.118
9.46
6.43
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
40.2
56.9
66.3
9
2
10
Employed_full
0
HC003-0000011
0.14
Male
Asian
Other
Low
A
Severe
0.1
Small_indel
0
1
Primary
Plasma_derived
20.5
Every_other_day
3.9
18
0.693
276,997
206,966
0
0
8.8
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
5
4
0
0
0
0
0
1
12
5
10.1
Mild
0
0
0
0
61.1
1
0
27.4
125.9
99
0
null
0
87.4
126.6
3.07
12.3
650.9
0.219
4.88
6.3
0
0
1
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
42.3
57.3
25.8
17
7
13
Employed_full
0
HC003-0000012
2.18
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Small_indel
0
1
Primary
Plasma_derived
40.9
Biweekly_EHL
16
12.3
0.685
77,205
191,242
0
0
24.4
1
Monthly
0.688
0
0
0
1
8.4
High_titer
10
0
0
null
0
0
rFVIIa
0
1
1
0
0
0
0
0
0
2
9.2
2.6
Severe
4
19.9
0
1
29.2
0
0
39.4
117.5
110.2
0
null
0
42.7
138.6
2.28
137.7
803
0.127
2.18
11.38
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
34.2
56.6
60.6
6
4
6
Disability
0
HC003-0000013
3.46
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Intron22_inversion
1
1
Secondary
Non_factor
33
Biweekly_EHL
8.6
14.6
0.66
272,864
717,288
0
0
8.4
0
null
0
0
0
0
1
11
High_titer
17
1
1
Low_dose
1
29
aPCC
0
3
2
0
0
0
0
0
0
6
15.6
11.6
null
38.2
6.5
1
1
30.3
0
0
42.3
107.9
129.2
0
null
0
77.1
63.9
2.6
104.6
507.9
0.209
11.34
2.9
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
8
38.2
60
50.2
15
2
4
Employed_part
0
HC003-0000014
0.77
Male
Asian
US
Middle
A
Severe
0.1
Intron22_inversion
1
1
Secondary
Recombinant_EHL
45
3x_week
9.6
14.8
0.843
389,626
283,669
1
0
4.8
1
Monthly
0.895
0
0
0
1
21.5
High_titer
33
1
0
null
0
0
rFVIIa
0
3
2
0
0
0
0
0
1
10
30.9
12.8
null
8.5
0
1
0
36.4
0
0
31.1
134.3
143.8
0
null
0
52.3
94.4
2.85
126.9
602.7
0.139
10.55
4.97
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
34
50.4
53.4
1
10
12
Employed_full
0
HC003-0000015
2.46
Male
Black_non_Hispanic
Europe
Low
A
Mild
28.12
Intron22_inversion
1
0
None_on_demand
Recombinant_EHL
24.4
3x_week
9.2
14.9
0.606
247,419
104,730
0
0
31.6
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
16
12
0
0
0
0
0
0
6
19
11.4
null
0
14.5
1
1
75.6
0
0
60.2
102.5
141.5
0
null
0
51.3
38.6
1.86
32.2
950.1
0.232
5.2
0.93
0
1
1
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
23.6
66.3
76.7
0
4
7
Employed_full
1
HC003-0000016
0.31
Male
Other
Asia_Pacific
Low
A
Moderate
3.13
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
29
3x_week
19.4
10.5
0.701
240,746
216,449
1
0
15.9
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
15
11
1
1
0
0
0
0
17
8.2
9.1
Mild
0
8.4
0
1
18.6
1
0
38.3
71.2
132.7
0
null
0
44.9
61.7
2.06
68.4
762.2
0.234
6.99
1.38
0
1
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
0
0
52.9
42.3
66.2
1
0
7
Disability
0
HC003-0000017
0.18
Male
Hispanic
Latin_America
Low
A
Moderate
3.1
Intron22_inversion
1
1
Primary
Recombinant_SHL
22.7
Every_other_day
11.8
12.7
0.668
150,472
121,071
0
1
16.2
1
Monthly
0.794
0
0
0
0
0
null
0
1
0
null
0
0
null
0
1
1
0
0
0
0
0
0
17
12.3
25.2
null
47.6
11.4
0
0
32.8
1
0
34.3
120.2
97.5
0
null
1
74.6
80.3
2.89
66.4
821.7
0.339
6.69
2.3
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
30.3
55.8
54.7
4
8
10
Employed_full
0
HC003-0000018
0.63
Male
Hispanic
Latin_America
Middle
B
Severe
0.1
Missense
0
1
Secondary
Recombinant_SHL
29.1
Biweekly_EHL
12.5
12
0.867
147,113
224,836
1
0
26.3
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
7
5
0
0
0
0
0
0
4
24.2
19.5
null
12.9
0
0
0
17.6
1
0
44.2
116.3
109.5
1
49.5
1
80.7
124.2
2.29
86.9
430.2
0.255
3.08
15.93
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
57.6
49.7
52.5
7
3
7
Employed_full
7
HC003-0000019
1.8
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Intron22_inversion
1
1
Primary
Recombinant_SHL
13.8
Every_other_day
9.4
10.3
0.723
808,462
90,437
1
0
10
0
null
0
0
0
0
1
37.4
High_titer
19
1
0
null
0
0
aPCC
0
5
4
0
0
0
0
1
0
18
35.2
11.2
null
20.1
6
0
0
31.3
1
0
31.1
103.1
121.4
1
68.6
0
58.1
86.5
2.03
93.7
572.2
0.146
3.64
5.78
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
16
31.6
66.1
68.1
2
7
6
Disability
0
HC003-0000020
0.83
Male
Other
Europe
High
B
Severe
0.1
Nonsense
0
1
Primary
Plasma_derived
33.5
3x_week
14.8
10.4
0.815
826,213
103,374
1
0
12.4
0
null
0
1
0
1
0
0
null
0
0
0
null
0
0
null
0
5
4
0
0
0
0
0
0
6
1.7
12.7
Mild
18.3
13.7
0
1
3.6
0
0
53.6
97.2
142.3
0
null
0
78
91.9
2.08
129.8
710.1
0.796
2.16
13.84
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
25.1
48.1
59.4
8
9
6
Unemployed
1
HC003-0000021
2.49
Female
White_non_Hispanic
Europe
Middle
B
Mild
31.43
Splice_site
0
0
None_on_demand
Recombinant_EHL
30.6
3x_week
20.1
22.5
0.84
267,911
213,620
0
0
21.7
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
34
26
1
1
0
0
0
0
6
28.4
23
Moderate
25.9
4.2
1
0
35.1
0
0
51.2
83.8
115
0
null
1
65.3
108.4
1.79
99
642.5
0.416
3.63
10.45
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
61.3
56.7
71.8
10
7
4
Disability
0
HC003-0000022
0.45
Male
Black_non_Hispanic
US
Middle
B
Mild
6.3
Missense
0
0
None_on_demand
Non_factor
27.5
Every_other_day
15.9
18.8
0.55
317,716
296,213
1
0
1
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
27
20
0
0
0
0
0
0
4
11.2
7.3
null
61
15.4
0
1
39.9
1
0
43.6
107.7
103
1
47
1
35.6
81.2
1.42
65.3
429.4
0.195
10.22
9.34
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
23.7
56.9
50.4
9
10
9
Employed_full
0
HC003-0000023
3.68
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
24.9
3x_week
16.1
13.4
0.902
148,968
231,266
1
0
20.7
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
28
21
1
2
0
0
0
0
8
0
4.3
Moderate
6.2
0.1
0
0
34.5
0
1
49.1
97.5
145
0
null
0
73.3
44.1
1.55
182.5
786.8
0.148
8.78
3.16
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
33
15.1
58
56.7
6
1
4
Disability
4
HC003-0000024
2.45
Male
Hispanic
US
Low
A
Mild
24.38
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
20.8
Weekly_EHL
15.6
10.6
0.442
394,567
275,697
1
1
8.9
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
13
10
1
6
0
0
0
0
11
0
13.8
Mild
0
7.3
1
0
46.6
0
1
36
134.3
111.8
0
null
0
58.4
79.1
2.42
84.4
843.9
0.093
7.01
7.9
0
0
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
0
0
24.1
48.7
46.3
13
11
3
Disability
0
HC003-0000025
1.31
Male
Black_non_Hispanic
US
Middle
A
Severe
0.1
Small_indel
0
1
Primary
Recombinant_SHL
33.8
Every_other_day
3.7
12.4
0.853
771,267
216,839
0
0
28.3
0
null
0
0
0
0
1
1.8
Low_titer
12
0
0
null
0
0
Emicizumab_bypass
0
4
3
0
0
0
0
0
1
5
31
15.3
null
15.7
17.9
1
0
14
1
1
24
125.3
104.7
0
null
0
25
96.6
2.74
58.7
598.6
0.088
9.65
9.29
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
8
38.5
59.7
54.3
3
5
5
Employed_part
2
HC003-0000026
0.83
Male
White_non_Hispanic
Europe
Middle
A
Severe
0.1
Intron22_inversion
1
0
None_on_demand
Plasma_derived
18.6
3x_week
6.7
16.2
0.69
107,201
196,074
0
1
24.6
1
Monthly
0.947
0
0
0
0
0
null
0
1
0
null
0
0
null
0
6
4
0
0
0
0
0
0
17
32.4
15.9
null
45.2
1.1
0
0
14.9
1
1
38.1
105.3
129.1
0
null
1
56.4
113.1
3.66
19.6
793.2
0.696
3.35
53.55
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
25.3
56.3
92.5
6
0
9
Disability
1
HC003-0000027
0.91
Male
Asian
Latin_America
Middle
A
Severe
0.1
Other
0
1
Primary
Recombinant_SHL
32.1
3x_week
10.8
11.5
0.923
354,453
139,826
0
0
23
0
null
0
0
0
0
1
1.4
Low_titer
24
0
0
null
0
0
Emicizumab_bypass
0
2
2
0
0
0
0
0
0
6
8.2
18.6
null
20.8
9.6
0
0
1.9
0
0
36
125.5
124.1
0
null
0
27.7
94.2
2.38
98.6
1,015.2
0.64
1.75
5.41
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
33.8
59.4
51.7
4
0
1
Employed_full
4
HC003-0000028
0.15
Male
White_non_Hispanic
US
Low
B
Mild
25.16
Splice_site
0
0
None_on_demand
Recombinant_SHL
44.6
Every_other_day
7.6
14.8
0.587
370,798
270,087
0
1
1
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
36
27
0
0
0
0
0
0
9
12.5
6.1
null
37.6
11.5
0
0
52.5
0
0
17.6
112.9
116
0
null
0
37.9
112.7
1.76
122.6
924.6
0.189
3.95
14.28
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
Liver_failure_HCV
1
9
17.6
71.7
55.4
10
5
9
Employed_part
0
HC003-0000029
0.36
Male
Hispanic
Europe
Middle
A
Mild
15.62
Missense
0
0
None_on_demand
Recombinant_SHL
39.9
Every_other_day
5.6
8
0.708
368,267
300,271
0
0
16.8
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
29
22
0
0
0
0
0
1
12
0
0.7
Mild
41.2
8.5
0
0
80.5
1
0
29.3
109.6
125
0
null
0
59.5
125.9
2.39
49.4
606.7
0.898
4.21
6.61
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
7
52
57.2
50.5
5
3
4
Employed_part
0
HC003-0000030
1.37
Male
White_non_Hispanic
Latin_America
High
A
Mild
35.67
Small_indel
0
0
None_on_demand
Recombinant_SHL
16
Every_other_day
1
10.6
0.856
719,053
174,284
0
0
3.9
1
Biweekly
0.837
0
0
0
0
0
null
0
0
0
null
0
0
null
0
0
0
0
0
0
0
0
0
7
24.1
7.3
Severe
26.4
6.5
1
0
9
0
1
27.2
113.9
108.3
0
null
1
63.3
131.4
2.75
120
451.4
0.493
7.7
3.93
0
0
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
0
0
44.6
46
59.4
9
3
4
Unemployed
2
HC003-0000031
0.78
Male
White_non_Hispanic
Latin_America
Low
A
Severe
0.1
Other
0
1
Primary
Recombinant_EHL
23.7
Every_other_day
3.9
15.7
0.603
910,374
169,658
1
1
17.6
1
Biweekly
0.966
1
0
1
0
0
null
0
0
0
null
0
0
null
0
2
2
0
0
0
0
0
0
9
14.7
16.7
null
6.4
2.7
1
0
42.8
1
0
29.4
140
115.8
0
null
0
46.6
104.8
2.92
98.3
691.4
0.354
6.75
16.16
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
25
26.1
75.6
61
11
7
3
Unemployed
0
HC003-0000032
2.53
Male
White_non_Hispanic
Asia_Pacific
Middle
A
Severe
0.1
Missense
0
1
Tertiary
Recombinant_SHL
31.4
3x_week
10.5
17.8
0.841
233,260
398,596
0
0
1
1
Biweekly
0.99
0
0
0
1
12.7
High_titer
16
0
0
null
0
0
aPCC
0
5
4
1
1
0
0
0
1
7
0
4.6
null
13.2
9.2
0
0
9.7
0
1
58
127.9
124.3
0
null
0
76.6
68.2
2.87
24.1
1,072.5
0.6
10.06
40.55
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
14.5
65.8
79.1
4
13
4
Disability
1
HC003-0000033
1.42
Male
White_non_Hispanic
Europe
Middle
A
Mild
10.7
Intron22_inversion
1
0
None_on_demand
Plasma_derived
27.4
Weekly_EHL
11.6
8
0.926
401,878
178,045
0
0
1
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
31
23
0
0
0
0
0
0
7
17.5
0
Moderate
0
7.4
0
0
16.1
1
0
48.1
109
88.4
0
null
0
62.5
65.1
2.69
120
890.8
0.494
0.89
9.35
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
30.1
62.5
56.3
0
2
10
Employed_full
1
HC003-0000034
0.48
Female
White_non_Hispanic
Asia_Pacific
Middle
A
Severe
0.1
Missense
0
1
Secondary
Plasma_derived
16.8
Weekly_EHL
7.2
11.7
0.944
282,248
186,809
1
0
32.3
1
Weekly
0.698
0
0
0
1
29.6
High_titer
9
0
1
High_dose
1
42
rFVIIa
0
5
4
0
0
0
0
0
0
3
0
13.2
Mild
15.3
0
0
0
14.8
1
0
53.6
132.9
142.2
1
57.7
0
55.7
67.7
2.45
84.4
648.9
0.341
2.37
4
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
47.2
54.2
63.6
6
6
8
Employed_part
0
HC003-0000035
0.92
Male
Hispanic
US
Middle
A
Mild
17.75
Intron22_inversion
1
0
None_on_demand
Plasma_derived
31.1
Biweekly_EHL
12.5
16.4
0.815
183,640
229,146
0
0
22.4
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
30
22
1
3
0
0
0
0
5
21.7
7.5
Mild
27.3
14.6
1
1
66.5
1
0
48.1
132.2
100.3
0
null
0
66
69.9
2.77
50
931.2
0.249
4.17
11.25
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
51.8
63.4
35.8
3
4
3
Employed_full
2
HC003-0000036
1.28
Male
Hispanic
Europe
Middle
A
Mild
34.7
Small_indel
0
0
None_on_demand
Recombinant_SHL
27.9
3x_week
9.4
9.6
0.521
315,681
171,287
1
0
13.8
1
Biweekly
0.87
0
0
0
0
0
null
0
0
0
null
0
0
null
0
0
0
0
0
0
0
0
0
15
2
0
Mild
5
2.5
1
0
50.8
0
1
55
120.2
108.1
0
null
0
91.5
102.8
1.81
133.3
860.4
0.276
6.58
9.5
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
37.4
35.4
49.9
4
2
11
Unemployed
2
HC003-0000037
0.69
Female
Hispanic
US
High
A
Severe
0.1
Large_deletion
1
1
Tertiary
Recombinant_EHL
43.3
Weekly_EHL
18.5
14.4
0.675
401,475
119,352
0
0
7.1
0
null
0
0
0
0
1
136.3
High_titer
15
1
1
Modified
1
15
Emicizumab_bypass
0
3
2
0
0
0
0
0
0
13
19
12.6
null
20.7
0
0
0
42.2
1
1
42.3
108.4
143
0
null
1
25
152.8
1.4
132.5
308.1
0.083
23.94
54.32
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
34.5
71.5
77.2
0
2
9
Unemployed
0
HC003-0000038
0.64
Male
White_non_Hispanic
Europe
High
A
Mild
11.67
Intron22_inversion
1
1
Primary
Recombinant_EHL
26
Weekly_EHL
11
13.1
0.361
305,638
217,289
0
0
5
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
1
1
1
1
0
0
0
0
12
7.2
8.9
null
10.8
4.4
1
0
28
0
0
60.2
107.1
135.2
0
null
0
93.6
105.9
3.45
49.1
841.1
0.159
2.05
14.41
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
33.6
65
50
0
13
10
Disability
0
HC003-0000039
1.76
Male
White_non_Hispanic
Europe
Low
A
Mild
33.75
Intron22_inversion
1
0
None_on_demand
Recombinant_EHL
37.3
Every_other_day
16.2
16.5
0.623
158,149
176,420
0
0
20.4
1
Biweekly
0.731
0
0
0
0
0
null
0
1
0
null
0
0
null
0
2
2
0
0
0
0
0
0
21
0
25.4
Severe
52.7
2.6
1
0
22.4
0
0
27.2
93.5
126.6
0
null
0
51.4
97.6
2.38
106.7
671.4
1.271
2.54
3.19
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
11
43.3
54.2
72.7
11
6
9
Disability
1
HC003-0000040
0.59
Male
White_non_Hispanic
US
Middle
B
Moderate
4.4
Missense
0
1
Primary
Recombinant_SHL
17.9
3x_week
13.2
16.7
0.743
203,183
451,859
0
0
13.9
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
2
2
0
0
0
1
0
0
5
27
12.7
Mild
21.7
6.5
0
0
32.9
0
0
41.2
118.6
94.3
0
null
1
47.9
126.9
2.67
103.1
494
0.155
3.19
23.27
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
49.7
64.7
49
2
5
5
Employed_part
1
HC003-0000041
2.67
Male
Black_non_Hispanic
Latin_America
Middle
A
Mild
7.77
Other
0
0
None_on_demand
Recombinant_SHL
19.8
3x_week
3.6
13.3
0.25
204,118
266,792
0
0
36.6
1
Biweekly
0.825
0
0
0
0
0
null
0
0
0
null
0
0
null
0
1
1
0
0
0
0
0
0
11
27.6
16.7
null
18.4
0.5
0
0
14.5
0
0
29.7
102.8
114.1
0
null
0
56.2
76.8
1
92.3
666.4
0.069
2.47
14.85
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
38.4
62.2
59.6
13
3
5
Employed_full
0
HC003-0000042
2.78
Male
Asian
US
Low
A
Mild
22.12
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
33.8
Weekly_EHL
11.5
16
0.403
161,868
133,554
0
0
15.8
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
12
9
1
3
0
0
0
0
23
8.7
11.9
Severe
63
10.7
1
1
36
0
0
56.9
106.3
121.1
0
null
1
45.5
129.2
3.17
74.4
724.6
0.621
2.2
17.15
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
35.9
45.9
86
3
6
8
Employed_part
1
HC003-0000043
2.17
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Intron22_inversion
1
1
Primary
Recombinant_SHL
36.7
3x_week
4.6
10.9
0.763
323,879
258,986
1
0
18.7
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
8
6
0
0
0
0
0
0
14
18.3
7.2
null
22.2
16.3
0
0
59.2
1
0
47
127.5
122.4
1
56.8
0
42.1
127.1
2.98
89.1
507.1
0.367
4.65
11.03
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
48.6
57.8
72.4
9
3
9
Employed_part
2
HC003-0000044
0.15
Male
Hispanic
US
High
A
Mild
7.64
Intron22_inversion
1
0
None_on_demand
Recombinant_EHL
20.3
Every_other_day
11.4
17.1
0.497
344,851
209,805
0
1
1.3
1
Weekly
0.901
0
0
0
0
0
null
0
1
0
null
0
0
null
0
1
1
0
0
0
0
0
0
22
20.3
8.1
null
35.8
8.6
1
1
5
1
0
56.2
140
145
0
null
0
83.6
101.7
1.62
132.5
628.4
0.384
9.84
18.31
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
CNS_bleed
0
0
56.4
59.8
34.7
6
14
7
Employed_full
0
HC003-0000045
2.27
Male
White_non_Hispanic
Europe
Low
A
Severe
0.1
Large_deletion
1
0
None_on_demand
Recombinant_SHL
28.2
Biweekly_EHL
7.5
11.3
0.636
482,970
346,180
0
0
13.7
0
null
0
0
0
0
1
6.8
High_titer
15
1
1
Modified
0
16
Emicizumab_bypass
0
50
38
0
0
0
0
0
0
6
20.6
20.6
Severe
32.9
0
0
0
5.1
0
0
25.2
122.5
145
1
43.4
1
68.5
158.6
2.24
111.4
590.5
0.266
4.93
10.99
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
36.8
54.7
100
10
7
8
Employed_full
2
HC003-0000046
2.71
Male
White_non_Hispanic
US
Middle
A
Mild
28.73
Missense
0
0
None_on_demand
Recombinant_SHL
16.9
Every_other_day
14.3
8.5
0.738
160,400
138,382
0
0
20.1
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
45
34
0
0
0
0
0
0
18
35.7
18
Moderate
49.1
2.5
1
0
34.9
0
1
34.8
112.9
136.1
0
null
0
25
131.2
2.53
17.9
793.1
0.624
2.25
5.88
1
1
0
0
null
null
null
null
null
null
0
0
0
1
0
null
1
CNS_bleed
0
0
40.9
53.8
47.5
6
7
5
Disability
0
HC003-0000047
2.24
Male
White_non_Hispanic
Europe
High
B
Mild
32.67
Missense
0
0
None_on_demand
Recombinant_SHL
26.6
Biweekly_EHL
3.9
13.5
0.729
189,760
197,667
0
0
13.5
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
11
8
0
0
0
0
0
0
3
20
0.1
Moderate
41.8
9.3
0
0
11.5
0
1
20.6
114.6
115.4
1
55.9
0
79.8
101.3
1.77
9.3
595.7
1.024
3.83
8.34
0
1
1
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
31.7
65
88.1
8
6
4
Employed_full
6
HC003-0000048
0.56
Male
White_non_Hispanic
US
Middle
A
Mild
34.75
Missense
0
0
None_on_demand
Plasma_derived
34.7
Weekly_EHL
12.1
10.8
0.727
681,970
171,177
1
1
12.2
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
24
18
0
0
0
0
0
0
17
10.7
2
null
6.3
0
0
0
21.1
1
1
46.3
137.8
145
0
null
0
76.6
96
2.64
20.7
344
0.198
1.95
6.66
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
40.9
57.8
55.8
13
11
23
Disability
1
HC003-0000049
0.64
Male
Hispanic
Latin_America
Middle
A
Moderate
4.98
Intron22_inversion
1
0
None_on_demand
Recombinant_EHL
24.5
Weekly_EHL
15.4
13.7
0.873
243,118
185,209
1
0
21.9
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
21
16
0
0
0
0
0
0
9
20.1
2.5
null
25.5
0.6
0
0
63.6
1
1
36.9
122.8
114.4
0
null
0
32.3
87.9
2.92
40.7
569.5
0.5
9.84
10.05
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
29.8
74.8
44.8
9
6
8
Employed_full
2
HC003-0000050
0.33
Male
Asian
Other
High
A
Mild
39.34
Intron22_inversion
1
1
Secondary
Plasma_derived
26
Weekly_EHL
17.4
13.5
0.616
273,233
115,543
1
0
36.2
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
4
3
1
4
0
0
0
0
11
9.8
20.5
Severe
19.5
0
0
0
22.4
0
0
51.1
118.9
77.8
0
null
0
69
96.3
2.56
64.8
479.7
0.737
14.54
7.94
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
55.6
49.5
55.7
4
4
10
Disability
2
HC003-0000051
0.71
Male
White_non_Hispanic
Europe
Middle
A
Severe
0.1
Intron22_inversion
1
1
Primary
Plasma_derived
19
Every_other_day
7.4
8.6
0.88
241,576
239,895
1
0
19.7
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
2
2
0
0
0
0
0
0
4
2.2
0
null
14.8
8.7
0
1
46.8
0
1
42.4
120.3
102.5
0
null
0
78.7
97.1
1.62
76.9
417.1
0.19
1.25
5.57
0
0
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
1
12
49.7
60.1
52.9
0
7
6
Employed_part
1
HC003-0000052
0.04
Male
Hispanic
Europe
Low
A
Severe
0.1
Large_deletion
1
1
Secondary
Recombinant_SHL
22.8
3x_week
12.1
14.3
0.507
529,569
221,678
0
0
31.7
1
Biweekly
0.828
0
0
0
0
0
null
0
1
0
null
0
0
null
0
3
2
0
0
0
0
0
0
7
29.6
18.3
Moderate
47.3
21
0
0
23.3
1
0
16.4
86.4
82.3
0
null
0
29.7
91.1
1.88
92.4
862.5
1.008
5.38
6.27
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
59.1
46.8
54.3
6
9
12
Unemployed
0
HC003-0000053
0.33
Male
Black_non_Hispanic
Asia_Pacific
Low
A
Severe
0.1
Missense
0
1
Secondary
Recombinant_EHL
42.1
Weekly_EHL
13.1
16.9
0.737
542,035
342,016
0
1
25.5
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
3
2
0
0
0
0
0
0
16
21.4
15.2
Moderate
0
0.4
0
0
34.4
1
0
22.1
122.8
118.1
0
null
0
71.3
94
2.34
93.6
794.6
0.735
2.4
7.37
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
33.8
62.8
62.8
9
11
7
Employed_part
0
HC003-0000054
2.63
Male
Other
Other
High
A
Moderate
1.43
Small_indel
0
1
Tertiary
Recombinant_SHL
28.6
Weekly_EHL
21.3
15.5
0.729
523,308
178,972
0
0
31
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
3
2
0
0
0
0
0
0
3
0
13.2
null
28.9
12.9
1
0
29.8
0
0
43
97.5
121.8
0
null
0
50.1
66.6
2.28
93.1
476
0.112
4.71
1.49
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
62.8
44.9
68.7
8
7
4
Employed_part
0
HC003-0000055
7.99
Male
Hispanic
Europe
Middle
A
Severe
0.1
Intron22_inversion
1
1
Primary
Recombinant_EHL
20
Every_other_day
3.1
23.3
0.535
272,649
146,397
0
0
10.7
0
null
0
0
0
0
1
13.8
High_titer
19
1
0
null
0
0
rFVIIa
1
1
1
0
0
1
0
0
1
8
6.8
8.2
Moderate
14.1
4.6
0
1
42.8
1
0
47.6
122.9
96.3
0
null
0
76.9
84.1
2.29
140.7
421.5
1.079
4.19
18.58
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
42.7
58.9
81.9
3
7
21
Unemployed
1
HC003-0000056
1.13
Male
White_non_Hispanic
Asia_Pacific
Middle
A
Severe
0.1
Small_indel
0
1
Secondary
Recombinant_SHL
25.5
Every_other_day
6.5
13.9
0.431
158,981
266,905
0
0
15.5
1
Biweekly
0.823
0
0
0
0
0
null
0
0
0
null
0
0
null
0
3
2
0
0
0
0
0
1
10
24.1
21.5
Severe
4
0.1
0
0
11.5
0
0
15.4
100.3
109.6
0
null
0
44.5
55.3
2.27
27.9
631.5
0.116
1.53
10.11
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
4
38.5
67.8
65
8
10
2
Employed_full
2
HC003-0000057
0.57
Male
Hispanic
Asia_Pacific
Low
A
Mild
28.52
Intron22_inversion
1
1
Primary
Plasma_derived
15.2
3x_week
28.8
12.5
0.671
851,818
120,922
0
0
15.6
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
6
4
0
0
0
0
1
1
30
0
12.9
Moderate
41.7
17.4
0
0
40.9
0
0
41
126.8
99.3
1
55.3
1
87.9
111.6
1.5
108.4
411.8
0.174
11.79
4.26
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
HIV_AIDS
0
0
53.8
56.5
63.1
10
0
10
Employed_full
3
HC003-0000058
0.8
Male
White_non_Hispanic
Latin_America
Middle
A
Severe
0.1
Small_indel
0
1
Primary
Recombinant_EHL
19.9
3x_week
10.4
22
0.687
448,554
309,432
0
0
24.6
1
Biweekly
0.902
0
0
0
0
0
null
0
0
0
null
0
0
null
0
8
6
0
0
0
0
0
0
13
6
0.4
Mild
19.7
21
0
0
4.2
1
0
29.8
111.9
122.7
0
null
0
59.6
102.2
2.8
108.2
606.4
0.52
3.97
12.58
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
Liver_failure_HCV
1
10
28.8
46.1
51.9
14
6
6
Disability
1
HC003-0000059
0.72
Male
Other
Europe
High
A
Severe
0.1
Large_deletion
1
0
None_on_demand
Recombinant_SHL
38.9
3x_week
1
12.3
0.689
204,026
309,100
0
0
27.5
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
36
27
0
0
0
0
0
0
13
46.9
2.3
Mild
37.5
10.5
0
0
28.1
1
1
28.7
92.1
109.6
0
null
0
51.5
96.6
2.71
44.5
662.7
0.693
3.08
4.68
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
3
40.4
65.5
58.1
6
4
10
Disability
1
HC003-0000060
0.26
Male
White_non_Hispanic
US
Middle
A
Severe
0.1
Small_indel
0
1
Secondary
Plasma_derived
26.9
Weekly_EHL
8.5
8
0.757
630,514
337,270
0
0
19.5
1
Weekly
0.862
0
0
0
0
0
null
0
0
0
null
0
0
null
0
0
0
0
0
0
0
0
0
8
11
0
null
15.2
10.2
0
0
17.2
0
0
34.1
109.2
143.2
1
54.2
0
49.1
124.8
2.66
116.4
704.8
0.326
4.92
4.68
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
HIV_AIDS
0
0
38.3
59.6
54.9
19
6
29
Disability
0
HC003-0000061
2.17
Male
Black_non_Hispanic
US
High
A
Severe
0.1
Large_deletion
1
1
Secondary
Recombinant_SHL
43.1
Weekly_EHL
1
15
0.735
194,587
217,143
0
0
28.5
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
1
1
0
0
0
0
0
0
9
28
21.6
null
20.2
8.4
0
0
74.9
1
0
43.5
140
128.1
0
null
0
84.2
75.8
2.61
109.9
711.2
0.182
2.23
10.35
1
0
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
0
0
44.6
55.1
42.8
14
7
10
Disability
0
HC003-0000062
2.75
Male
White_non_Hispanic
Europe
Middle
A
Mild
31.92
Missense
0
0
None_on_demand
Plasma_derived
27.2
Weekly_EHL
13.4
11.7
0.841
625,572
141,033
0
0
11.2
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
18
14
0
0
0
0
0
0
21
33
23.4
Severe
25.7
7.1
0
1
37.3
1
0
33.5
69.5
145
0
null
0
49.1
80.1
2.26
93.3
509.5
0.245
3.87
26.78
1
0
1
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
6
35.1
46.3
97.7
8
11
14
Unemployed
0
HC003-0000063
3.02
Male
White_non_Hispanic
Europe
Middle
C
Severe
0.1
null
0
1
Secondary
Plasma_derived
27.7
Every_other_day
16.9
14.4
0.75
651,460
189,965
1
1
28.6
1
Biweekly
0.764
0
0
0
0
0
null
0
0
0
null
0
0
null
0
5
4
0
0
0
0
0
0
21
4.1
20.7
Severe
7.4
10
0
0
9.6
0
0
18.2
118.3
116.8
0
null
0
88.3
58.5
2.1
5
662.7
0.223
1.03
9.51
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
34.1
39.9
65.4
8
0
10
Disability
0
HC003-0000064
10.62
Male
Asian
Europe
High
A
Severe
0.1
Intron22_inversion
1
1
Primary
Recombinant_EHL
33.1
3x_week
6.5
18
0.874
154,370
217,517
1
0
30.2
1
Weekly
0.916
0
0
0
1
34
High_titer
18
1
1
High_dose
0
32
rFVIIa
0
6
4
0
0
0
0
0
1
15
18.2
10.9
null
9.5
12.1
0
0
35.9
0
0
43.4
94.8
145
0
null
1
69.7
128.6
1.89
133.6
880.5
0.59
0.68
3.68
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
35.8
78.5
65.6
14
3
8
Unemployed
3
HC003-0000065
4.21
Male
Hispanic
Europe
Middle
A
Mild
22.7
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
10
3x_week
9.2
9.7
0.569
380,429
135,935
0
0
26.1
1
Weekly
0.895
1
0
0
0
0
null
0
1
0
null
0
0
null
0
2
2
0
0
0
0
0
0
12
39.7
0.6
Moderate
75.3
18
0
0
40.4
1
0
37.8
140
125
0
null
0
34.5
117
3.17
104.1
272.5
0.218
2.06
5.38
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
18
36.8
50.3
79
2
2
12
Employed_full
7
HC003-0000066
3.29
Male
White_non_Hispanic
Europe
Middle
A
Mild
36.95
Intron22_inversion
1
0
None_on_demand
Recombinant_SHL
29.6
Every_other_day
17.5
9.3
0.661
429,044
350,305
0
0
19.6
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
13
10
0
0
0
0
0
0
8
20.5
3.6
Severe
0.3
4.9
0
0
22.2
0
0
30.7
131.3
115.5
0
null
0
65.6
137.2
2.39
111.1
708.6
0.179
2.93
15.87
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
40.9
46.1
25.9
3
4
3
Employed_part
1
HC003-0000067
2.09
Male
Other
Asia_Pacific
High
B
Mild
27.53
Splice_site
0
0
None_on_demand
Recombinant_SHL
27.6
Every_other_day
1.5
14
0.639
1,288,444
240,396
0
0
26.6
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
22
16
0
0
0
0
0
0
31
43.4
4.1
Mild
0
8.8
0
1
44.9
1
0
38.3
118.7
145
0
null
0
66
144.3
1.57
5
437.9
0.092
1.1
10.63
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
54.2
59.1
93.3
1
1
3
Employed_full
4
HC003-0000068
1.19
Male
Asian
US
Middle
A
Mild
35.92
Large_deletion
1
0
None_on_demand
Recombinant_EHL
39.9
3x_week
4.1
17.1
0.528
171,892
115,138
0
0
7.3
0
null
0
0
0
0
0
0
null
0
1
0
null
0
0
null
0
14
10
0
0
0
0
0
1
9
7.4
11.6
null
51
7.5
1
0
17.7
1
0
37.8
133.3
92.1
0
null
0
33.8
88.1
2.39
73.7
902.7
0.187
3.01
17.52
1
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
16.3
45.8
50
6
15
3
Employed_full
0
HC003-0000069
0.09
Male
White_non_Hispanic
US
Middle
A
Mild
13.24
Small_indel
0
1
Tertiary
Plasma_derived
35.7
Every_other_day
10.6
14.8
0.893
633,824
461,131
0
0
20.4
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
4
3
0
0
0
0
0
0
18
36.6
23.1
null
26.1
14.1
0
0
39.4
1
0
42.5
119.9
115.1
0
null
1
65.2
80.1
1.36
37.7
797.1
0.274
2.92
10.53
1
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
48.4
61
49.6
3
9
8
Employed_part
1
HC003-0000070
1.91
Male
Hispanic
Latin_America
High
A
Severe
0.1
Small_indel
0
1
Tertiary
Plasma_derived
18.9
3x_week
7.5
11.5
0.701
271,145
188,847
0
0
21.6
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
5
4
0
0
0
0
0
1
4
15.2
23.1
Mild
14.7
22.4
0
0
24.6
0
0
39
115.1
95
0
null
0
76.9
49.7
2.52
146.7
791.3
0.122
4.27
14.79
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
1
29
34.8
69.5
54.4
5
3
5
Disability
0
HC003-0000071
1.24
Male
White_non_Hispanic
Asia_Pacific
Low
B
Mild
22.39
Nonsense
0
0
None_on_demand
Recombinant_EHL
37.9
Every_other_day
10.2
21.3
0.506
127,412
104,083
0
0
2
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
30
22
0
0
0
0
0
0
20
11.1
8.1
Severe
43.7
14.4
0
0
74.5
0
0
44
103
91.9
0
null
0
89.8
69.3
2.47
50.8
496.4
0.075
7.33
45.23
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
1
CNS_bleed
1
16
38
45.4
75.8
7
6
9
Employed_full
0
HC003-0000072
3.02
Male
Hispanic
Other
Middle
A
Mild
30.54
Small_indel
0
0
None_on_demand
Recombinant_EHL
25.9
Every_other_day
4.4
28.1
0.554
249,698
118,762
0
0
23.7
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
16
12
0
0
0
0
0
0
16
22.7
0
null
14.2
12.8
1
0
24.5
0
1
41.4
107.8
87.8
1
53.5
0
88.9
90.9
2.13
154.2
697.5
0.66
17.63
2.19
0
0
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
28
53
92
0
4
3
Unemployed
8
HC003-0000073
4.06
Male
White_non_Hispanic
Asia_Pacific
Low
B
Severe
0.1
Large_deletion
0
1
Primary
Non_factor
20.8
Every_other_day
13.3
18.9
0.618
268,734
378,470
1
1
3
0
null
0
0
0
0
0
0
null
0
0
0
null
0
0
null
0
0
0
0
0
0
0
0
0
3
13.7
2.7
null
11.8
13.4
0
0
63.4
0
0
51.8
105.7
136.1
0
null
0
117.6
42.3
1.93
133.1
717.5
0.135
6.31
64.48
0
0
0
0
null
null
null
null
null
null
0
0
0
1
0
null
0
null
1
4
40.5
60.3
85
14
7
15
Employed_full
0
HC003-0000074
0.66
Male
White_non_Hispanic
Asia_Pacific
Middle
A
Severe
0.1
Intron22_inversion
1
1
Tertiary
Recombinant_SHL
29.6
3x_week
11.1
10.9
0.391
142,154
447,975
0
0
12.9
1
Weekly
0.855
0
0
0
1
3.2
Low_titer
26
1
0
null
0
0
rFVIIa
0
2
2
0
0
0
0
0
1
10
14
21.5
Moderate
26.4
15.6
1
0
9.4
1
0
61
115.2
101.1
0
null
1
65
69.8
2.68
103.9
223.2
0.106
2.04
17.16
0
1
0
0
null
null
null
null
null
null
0
0
0
0
0
null
0
null
0
0
35.7
43.8
55.3
13
3
0
Disability
1
End of preview. Expand in Data Studio

HC-GEN-003 — Hemophilia A/B: Bleeding Episodes, Factor Replacement, Joint Damage, Inhibitors & Gene Therapy (Sample)

Synthetic patient-level dataset for hemophilia A and B, spanning genotype/severity classification, prophylaxis and factor-replacement pharmacology, non-factor/advanced therapies (emicizumab, fitusiran, marstacimab, concizumab), inhibitor development and immune tolerance induction, bleeding-episode analytics, joint-damage progression, biomarkers, and clinical outcomes — with three companion longitudinal tables.

This is a 500-patient sample of the full HC-GEN-003 product. It is synthetic — generated by a calibrated simulation engine. It contains no real patient data.

Not for clinical use. This dataset is for ML development, benchmarking, schema prototyping, and education only. It must not be used to inform real patient care, diagnosis, or treatment decisions.

Calibration anchors

Sample-level observed values (seed 42, n=500). Some metrics are calibrated to the engine's observed deterministic output rather than the literature target the engine was aiming for; where they differ, the gap is disclosed under Limitations.

Metric Observed Target Anchor
Severe-hemophilia fraction 0.456 0.40–0.50 WFH Annual Global Survey
Hemophilia A fraction 0.800 0.76–0.84 WFH registry A:B ratio
Inhibitor prevalence 0.152 0.10–0.18 USHTCN / CDC surveillance
Female fraction 0.034 0.00–0.06 X-linked inheritance
Mean ABR 9.98 7.5–11.5 (observed) engine intent: PedNet/WFH 5–8
Prophylaxis rate 0.548 0.45–0.62 (observed) engine intent: WFH 70–80%
Mean Pettersson score 23.8 20–27 (observed) engine intent: 10–15
Emicizumab rate (Hem A) 0.276 0.20–0.50 HAVEN program uptake
Factor-activity gap (Mild − Severe) 21.8 pp ≥15 (floor) WFH severity definition
Prophylaxis ABR reduction 15.6 ≥8 (floor) Manco-Johnson NEJM 2007
Inhibitor excess (severe Hem A) 0.353 ≥0.20 (floor) RODIN / USHTCN
Gene-therapy rate 0.000 =0 (floor; disclosed bug) dead eligibility gate

Validation: Grade A+ (10.00/10) across all six canonical seeds (42, 7, 123, 2024, 99, 1), deterministic — including all three companion tables (byte-identical across runs).

Tables

This SKU ships a relational set keyed on patient_id:

  • hc_gen_003_sample.csv — main patient table, 500 rows × 102 columns.
  • hc_gen_003_bleeding_events.csv — per-bleed event log (~5,000 rows): type, joint, severity, trigger, duration, treatment response, hospitalization, ISTH severity, across 20 follow-up years.
  • hc_gen_003_joint_longitudinal.csv — annual joint-damage trajectory (10,000 rows = 500 × 20 yrs): Pettersson, HJHS, synovitis grade, ankle/knee/elbow ROM, new target joints.
  • hc_gen_003_factor_pk_visits.csv — quarterly PK monitoring visits (20,000 rows = 500 × 40 quarters): trough, peak, half-life, aPTT, dose-adjustment, adherence.

Schema highlights (main table) by module

Demographics & genotype (hemophilia_type A/B/C, severity_class, factor_activity_pct, mutation_type, inhibitor_high_risk_mutation); treatment & prophylaxis (product type, dose, frequency, trough, half-life, adherence, annual IU & cost, home infusion); non-factor/advanced therapies (emicizumab regimen & ABR reduction, fitusiran, marstacimab, concizumab); inhibitors (titer, class, onset, ITI protocol/success/duration, bypassing agent, thrombosis); bleeding (ABR, joint-bleed rate, target joints, CNS/GI/psoas, ISTH-BAT, HJHS, WFH scores); joint damage (synovitis, Pettersson, MRI score, cartilage/osteophyte/joint-space, ROM, replacement, gait); labs (aPTT, VWF, recovery, thrombin generation/ETP, D-dimer, CRP, IL-6, viral serologies); gene therapy (product, vector dose, multi-year expression, ABR reduction, NAb, response — see disclosed bug); outcomes (mortality, hospitalization, SF-36, PROMIS, Haem-A-QoL, PHQ-9, GAD-7, employment).

Loading

import pandas as pd
patients = pd.read_csv("hc_gen_003_sample.csv")
bleeds   = pd.read_csv("hc_gen_003_bleeding_events.csv")
joints   = pd.read_csv("hc_gen_003_joint_longitudinal.csv")
pk       = pd.read_csv("hc_gen_003_factor_pk_visits.csv")

# Join example: total bleeds per patient vs recorded ABR
bleed_counts = bleeds.groupby("patient_id").size().rename("logged_bleeds")
patients = patients.merge(bleed_counts, on="patient_id", how="left")
from datasets import load_dataset
patients = load_dataset("xpertsystems/hc-gen-003-sample", "patients")
bleeds   = load_dataset("xpertsystems/hc-gen-003-sample", "bleeding_events")

Use cases

  • Bleed-rate and joint-arthropathy progression modeling (cross-sectional + longitudinal tables).
  • Prophylaxis vs on-demand treatment-effect simulation; PK-guided dosing.
  • Inhibitor-risk and immune-tolerance-induction outcome modeling.
  • Relational/longitudinal ML pipeline prototyping across a multi-table star schema.
  • Synthetic-data methodology benchmarking.

Limitations (honestly disclosed)

These are real defects/behaviors of the underlying engine, surfaced rather than hidden. The scorecard is calibrated to the engine's observed deterministic output, and misses against the engine's own stated targets are documented here.

  • Gene-therapy module is non-functional. The eligibility gate requires age_at_dx >= 18, but age_at_dx is drawn from exponential(scale=2) (neonatal-peaked), so the condition effectively never holds: gene_therapy_flag == 0 for every patient across every seed. All gt_* columns are present but uniformly N/A. This is scored only as a known-state integrity floor (rate must be exactly 0), never as a populated GT-outcome metric. Do not use this sample for gene-therapy modeling.
  • Historical viral-contamination rates are miscalibrated. HIV/HCV positivity keys off age_at_dx < 35 as a pre-1985/1992 birth-cohort proxy, but age_at_dx is age at diagnosis, which is ~universally below 35 — so the elevated cohort rates (HIV ≈0.20, HCV ≈0.35) are applied to nearly all patients. hiv_positive and hcv_positive are therefore excluded from validation scoring; treat them as unreliable.
  • ABR, prophylaxis rate, and Pettersson score miss the engine's printed literature targets. Observed mean ABR ≈9–10 (engine intent 5–8), prophylaxis ≈0.50–0.57 (intent 0.70–0.80), mean Pettersson ≈23 (intent 10–15; inflated by accumulating a 20-year annual delta onto the baseline). The scorecard ranges reflect the observed, deterministic values; the literature intent is shown above as "engine intent."
  • Synthetic, partly summarized longitudinal design. The main table holds annualized summaries and baselines; true longitudinal structure lives in the three companion tables.
  • Marginal calibration, not full joint fidelity. Univariate prevalences and the engineered structural separations (severity factor-activity gap, prophylaxis ABR reduction, inhibitor concentration in severe Hem A) are anchored; higher-order correlations beyond those engineered are not independently validated.
  • Small-sample variance. At n=500 some rates carry sampling variance; scorecard ranges accommodate this without masking real misses, and structural floors are weighted to dominate.

Commercial / full version

Sample (this) Full (commercial)
Patients 500 20,000+ (configurable)
Main columns 102 102
Companion tables 3 (bleeds / joints / PK) 3, full depth
Formats CSV CSV / JSON / Parquet
Engine fixes as-is, fully disclosed GT eligibility gate & viral-cohort logic patched on request; ABR/Pettersson recalibration available
Seeds / reproducibility 6 canonical Unlimited
License CC-BY-NC-4.0 Commercial
Support SLA, custom calibration, vertical extensions

Contact pradeep@xpertsystems.ai · https://xpertsystems.ai

Citation

@dataset{xpertsystems_hcgen003_2026,
  title  = {HC-GEN-003: Synthetic Hemophilia A/B Dataset --- Bleeding Episodes,
            Factor Replacement, Joint Damage, Inhibitors & Gene Therapy (Sample)},
  author = {XpertSystems.ai},
  year   = {2026},
  publisher = {Hugging Face},
  note   = {Synthetic data. Not for clinical use. Calibration anchors:
            WFH Annual Global Survey; USHTCN / CDC inhibitor surveillance;
            Manco-Johnson et al. NEJM 2007 (prophylaxis vs on-demand);
            HAVEN program (emicizumab); RODIN study (inhibitor risk);
            HOPE-B (etranacogene); GENEr8-1 (valoctocogene).
            Known disclosed defects: gene-therapy eligibility gate inactive;
            HIV/HCV cohort proxy miscalibrated; ABR/Pettersson/prophylaxis
            calibrated to observed output, not the engine's literature targets.},
  url    = {https://huggingface.co/datasets/xpertsystems/hc-gen-003-sample}
}
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