Table 1 Baseline characteristics and patient disposition

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 (%)

 ≤ 70^a

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)

 Withdrawal^b

4 (7.3)

 Lost to follow-up^c

2 (3.6)

 Other, enrolled into phase 2/3 idursulfase-IT trial (HGT-HIT-094, NCT02055118)

25 (45.5)