Table 2 Demographics and baseline characteristics
|  | Idursulfase-IT (n = 34) | No idursulfase-IT (n = 15) | Overall (N = 49) |
|---|---|---|---|
Age, years | |||
Mean (SD) | 5.0 (1.5) | 5.3 (2.6) | 5.1 (1.9) |
Median (range) | 4.6 (3.1, 8.7) | 4.8 (3.1, 13.0) | 4.6 (3.1, 13.0) |
Patients by age group, n (%) | |||
Aged < 6 years | 28 (82.4) | 12 (80.0) | 40 (81.6) |
Aged ≥ 6 years | 6 (17.6) | 3 (20.0) | 9 (18.4) |
Race, n (%) | |||
White | 23 (67.6) | 12 (80.0) | 35 (71.4) |
Asian | 4 (11.8) | 0 | 4 (8.2) |
Black or African American | 1 (2.9) | 0 | 1 (2.0) |
Other | 6 (17.6) | 3 (20.0) | 9 (18.4) |
Height, cm | |||
Mean (SD) | 111.7 (9.5) | 110.9 (11.9) | 111.5 (10.2) 108.8 (93.0, 140.0) |
Median (range) | 109.4 (95.7, 140.0) | 107.9 (93.0, 137.7) | |
Weight, kg | |||
Mean (SD) | 24.5 (4.9) | 25.3 (8.4) | 24.8 (6.1) |
Median (range) | 23.8 (18.5, 39.8) | 23.3 (17.0, 48.4) | 23.6 (17.0, 48.4) |
Baseline DAS-II GCA score | |||
Mean (SD) | 68.4 (8.3) | 67.3 (7.5) | 68.0 (8.0) |
Median (range) | 67.5 (55, 85) | 66.0 (56, 78) | 67.0 (55, 85) |
Patients by baseline DAS-II GCA score category, n (%) | |||
DAS-II GCA score ≤ 70 | 20 (58.8) | 9 (60.0) | 29 (59.2) |
DAS-II GCA score > 70 | 14 (41.2) | 6 (40.0) | 20 (40.8) |
Patients by type of IDS variant, n (%) | |||
Missense | 17 (50.0) | 7 (46.7) | 24 (49.0) |
Frameshift | 5 (14.7) | 3 (20.0) | 8 (16.3) |
Large deletion or complete deletion/large rearrangement | 5 (14.7) | 0 | 5 (10.2) |
Intronic | 2 (5.9) | 2 (13.3) | 4 (8.2) |
Nonsense | 3 (8.8) | 1 (6.7) | 4 (8.2) |
Splice site | 1 (2.9) | 0 | 1 (2.0) |
Unclassifiable | 1 (2.9) | 2 (13.3) | 3 (6.1) |