Table 2 Baseline characteristics and patient disposition, split according to study completion status
Characteristic/disposition | Completed study (n = 23) | Discontinued study (n = 32) |
|---|---|---|
Male, n (%) | 23 (100) | 32 (100) |
Age, years | ||
Mean (SD) | 7.30 (3.694) | 4.39 (2.411) |
Median (min, max) | 6.22 (3.4, 16.7) | 3.49 (2.0, 12.4) |
Age subgroups, n (%) | ||
< 7 years | 15 (65.2) | 27 (84.4) |
≥ 7 years | 8 (34.8) | 5 (15.6) |
Race, n (%) | ||
Asian | 1 (4.3) | 3 (9.4) |
Black or African American | 0 (0) | 1 (3.1) |
White | 20 (87.0) | 27 (84.4) |
Other | 2 (8.7) | 1 (3.1) |
Height, cm | ||
Mean (SD) | 117.6 (13.7) | 107.2 (14.4) |
Median (min, max) | 114.0 (101.0, 152.0) | 104.1 (85.6, 143.4) |
Weight, kg | ||
Mean (SD) | 28.3 (14.0) | 22.6 (6.3) |
Median (min, max) | 25.2 (16.0, 83.3) | 21.2 (14.6, 37.5) |
Genotype category, n (%) | ||
Null variant (frameshift, nonsense, premature truncation, and large deletion) | 2 (8.7) | 10 (31.3) |
Missense/presumed missense | 14 (60.9) | 19 (59.4) |
Splice site variant/presumed splice site variant | 7 (30.4) | 3 (9.4) |
Phenotype category, n (%) | ||
Mild | 9 (39.1) | 3 (9.4) |
Intermediate | 4 (17.4) | 2 (6.3) |
Severe | 7 (30.4) | 25 (78.1) |
Unclassifiable | 3 (13.0) | 2 (6.3) |
Baseline DAS-II GCA score, n (%) | ||
≤ 70 | 7 (30.4) | 11 (34.4) |
> 70 | 14 (60.9) | 12 (37.5) |
Missing | 2 (8.7) | 9 (28.1) |
Receiving IV idursulfase, n (%) | ||
Yes | 23 (100) | 32 (100) |
No | 0 (0) | 0 (0) |