Characteristic | Caregiver-reported patients (3–26 years) n = 31 | Self-reported patients (12–58 years) n = 20 |
---|---|---|
Age category, n (%) | ||
3 to < 12 years | 17 | 0 |
≥ 12 years | 14 | 16b |
Age in years, mean (SD) | 10.8 (6.0) | 22.5 (11.4) |
Male, n (%) | 31 (100) | 20 (100) |
Race, n (%)a | ||
White | 27 (87.1) | 16 (80.0) |
Black/African American | 4 (12.9) | – |
Unknown | – | 4 (20.0) |
With MPS-affected siblings, n (%) | 3 (9.7) | 4 (20.0) |
Age at diagnosis in years, mean (SD) | 2.7 (1.5) | 4.6 (3.0)c |
Type of MPS II | ||
Severe (with CNS involvement) | 20 (64.5) | 1 (5) |
Attenuated (without CNS involvement) | 10 (32.3) | 15 (75) |
Unknown | 1 (3.2) | 4 (20.0) |
Comorbidities, n (%) | ||
Cachexia | 1 (3.2) | 0 |
Cardiac disease | 17 (54.8) | 11 (55.0) |
Carpal tunnel syndrome | 19 (61.3) | 15 (75.0) |
Convulsions | 2 (6.5) | 0 |
Diarrhea | 7 (22.6) | 1 (5.0) |
Neurodegeneration | 6 (19.4) | 1 (5.0) |
Psychosocial problems | 14 (45.2) | 2 (10.0) |
Severity of CI, n (%) | 24 (77.4) | 1 (5.0)d |
Mild | 4 (16.7) | 0 |
Moderate | 9 (37.5) | 1 (5.0) |
Severe | 11 (45.8) | 0 |