Characteristic/disposition | Study population (N = 55) |
---|---|
Male, n (%) | 55 (100) |
Age, years | |
Mean (SD) | 5.60 (3.316) |
Median (min, max) | 4.82 (2.0, 16.7) |
Age subgroups, n (%) | |
< 7 years | 42 (76.4) |
≥ 7 years | 13 (23.6) |
Race, n (%) | |
Asian | 4 (7.3) |
Black or African American | 1 (1.8) |
White | 47 (85.5) |
Other | 3 (5.5) |
Height, cm | |
Mean (SD) | 111.58 (14.923) |
Median (min, max) | 110.10 (85.6, 152.0) |
Weight, kg | |
Mean (SD) | 25.00 (10.514) |
Median (min, max) | 22.50 (14.6, 83.3) |
Genotype category, n (%) | |
Null variant (frameshift, nonsense, premature truncated, and large deletion) | 12 (21.8) |
Missense/presumed missense | 33 (60.0) |
Presumed splice site variant/splice site variant | 10 (18.2) |
Baseline DAS-II GCA score, n (%) | |
≤ 70a | 18 (32.7) |
> 70 | 26 (47.3) |
Missing | 11 (20.0) |
Receiving IV idursulfase, n (%) | |
Yes | 55 (100) |
No | 0 (0) |
Patient disposition, n (%) | |
Completed | 23 (41.8) |
Discontinued | 32 (58.2) |
Reasons for discontinuation | |
Noncompliance with study procedure | 1 (1.8) |
Withdrawalb | 4 (7.3) |
Lost to follow-upc | 2 (3.6) |
Other, enrolled into phase 2/3 idursulfase-IT trial (HGT-HIT-094, NCT02055118) | 25 (45.5) |