Table 2 HS-FOCUS mean total scores by age and MPS II severity for caregiver- and self-reported patients
Caregiver-reported patients (3–26 years) | Self-reported patients (12–58 years) | ||||
|---|---|---|---|---|---|
Initial assessment at baseline | Follow-up at 2–4 weeks | Initial assessment at baseline | Follow-up at 2–4 weeks | ||
Age category | |||||
Pediatric/adolescent | Patients, n | 27 | 24 | 7 | 5 |
Mean (SD) total score | 0.92 (0.66) | 0.80 (0.51) | 0.50 (0.52) | 0.45 (0.62) | |
Adult | Patients, n | 4 | 4 | 13 | 13 |
Mean (SD) total score | 2.21 (0.67) | 1.95 (0.76) | 0.53 (0.67) | 0.47 (0.51) | |
MPS II severity | |||||
With CNS involvement | Patients, n | 20 | 18 | 1 | 1 |
Mean (SD) total score | 1.35 (0.81) | 1.20 (0.72) | 2.1 (−) | 1.55 (−) | |
Without CNS involvement | Patients, n | 10 | 9 | 15 | 14 |
Mean (SD) total score | 0.55 (0.43) | 0.51 (0.26) | 0.41 (0.53) | 0.44 (0.50) | |