Characteristic | Overall population (N = 61) | Prospective patients (n = 44) | Retrospective patients (n = 17) |
---|---|---|---|
Age at HOS entry | |||
 Number of patients with data available | 61 | 44 | 17 |
 Mean (SD), years | 13.5 (7.0) | 13.6 (7.8) | 13.4 (4.5) |
 Median (P10, P90), years | 13.2 (5.1, 22.4) | 13.4 (4.6, 23.2) | 13.0 (9.5, 19.4) |
Age at onset of signs and symptoms | |||
 Number of patients with data available | 55 | 38 | 17 |
 Mean (SD), years | 2.9 (3.2) | 3.1 (3.3) | 2.6 (2.8) |
 Median (P10, P90), years | 2.5 (0.2, 5.5) | 2.5 (0.3, 5.8) | 1.7 (0.1, 5.5) |
Age at diagnosis | |||
 Number of patients with data available | 56 | 39 | 17 |
 Mean (SD), years | 5.3 (5.4) | 5.6 (6.0) | 4.6 (3.6) |
 Median (P10, P90), years | 3.5 (1.2, 11.9) | 3.4 (1.2, 13.3) | 3.6 (0.1, 10.8) |
Ethnicity | |||
 Asian, n/N (%) | 59/60 (98.3) | 43/44 (97.7) | 16/16 (100) |
 Other, n/N (%) | 1/60 (1.7) | 1/44 (2.3) | 0/16 (0) |
Family history of MPS II, yes, n/N (%) | 28/50 (56.0) | 18/35 (51.4) | 10/15 (66.7) |
Sex, male, n/N (%) | 61/61 (100) | 44/44 (100) | 17/17 (100) |
Treated with idursulfase at any time, yes, n/N (%) | 24/49 (49.0) | 24/38 (63.2) | 0/11 (0) |
Age at idursulfase treatment start | |||
 Number of patients with data available | 24 | 24 | N/A |
 Mean (SD), years | 13.4 (9.2) | 13.4 (9.2) |  |
 Median (P10, P90), years | 12.5 (4.5, 24.9) | 12.5 (4.5, 24.9) |  |
Length of time on idursulfase treatment | |||
 Number of patients with data available | 24 | 24 | N/A |
 Mean (SD), months | 43.8 (27.0) | 43.8 (27.0) |  |
 Median (P10, P90), months | 41.6 (9.3, 79.3) | 41.6 (9.3, 79.3) |  |
Cognitive impairment at any time, yes, n/N (%) | 40/56 (71.4) | 24/39 (61.5) | 16/17 (94.1) |
Age at last visit in HOS | |||
 Number of patients with data available | 44 | 44 | N/A |
 Mean (SD), years | 16.9 (8.2) | 16.9 (8.2) |  |
 Median (P10, P90), years | 17.0 (7.3, 25.0) | 17.0 (7.3, 25.0) |  |
Deceased, yes, n/N (%) | 25/61 (41.0) | 8/44 (18.2) | 17/17 (100) |
Age at death | |||
 Number of patients with data available | 25 | 8 | 17 |
 Mean (SD), years | 14.4 (4.6) | 16.4 (4.3) | 13.4 (4.5) |
 Median (P10, P90), years | 13.4 (9.5, 19.4) | 16.7 (9.8, 24.1) | 13.0 (9.5, 19.4) |