ERT | HSCT | UP | |||||||||
---|---|---|---|---|---|---|---|---|---|---|---|
All | Pediatrics < 16 years old | Adults ≥ 16 years old | < 5 years on ERT | ≥ 5 years on ERT | All | Pediatrics < 16 years old | Adults ≥ 16 years old | All | Pediatrics < 16 years old | Adults ≥ 16 years old | |
N | 26 | 6 | 20 | 13 | 13 | 7 | 4 | 3 | 18 | 2 | 16 |
Sex | |||||||||||
Males | 15 | 5 | 10 | 9 | 6 | 3 | 1 | 2 | 8 | 0 | 8 |
Females | 11 | 1 | 10 | 4 | 7 | 4 | 3 | 1 | 10 | 2 | 8 |
Age at the time of survey | |||||||||||
Median | 21.1 | 12.7 | 29.8 | 18.6 | 29.3 | 13.6 | 12.2 | 20.7 | 29.2 | 11.8 | 30.0 |
Mean ± SD | 24.9 ± 10.8 | 12.8 ± 2.4 | 28.5 ± 9.6 | 22.0 ± 10.8 | 27.8 ± 10.3 | 15.9 ± 4.9 | 12.4 ± 0.9 | 20.7 ± 3.4 | 26.4 ± 7.8 | 11.8 ± 0.1 | 28.3 ± 6.1 |
Range | 10.3–45.7 | 10.3–15.7 | 16.3–45.7 | 10.3–42.2 | 13.6–45.7 | 11.4–24.2 | 11.4–13.6 | 17.3–24.2 | 11.7–38.4 | 11.7–11.8 | 17.3–38.4 |
Age symptoms were first noticed | |||||||||||
Median | 1.5 | 2.4 | 1.3 | 2.0 | 1.1 | 0.7 | 1.8 | 0.5 | 2.4 | 6.5 | 2.3 |
Mean ± SD | 2.6 ± 4.5 | 2.6 ± 2.2 | 2.7 ± 5.0 | 2.2 ± 1.5 | 3.0 ± 6.3 | 2.4 ± 4.0 | 3.7 ± 5.2 | 0.6 ± 0.3 | 5.8 ± 7.2 | 6.5 ± 5.6 | 5.7 ± 7.6 |
Range | 0.0–23.5 | 0.0–5.1 | 0.0–23.5 | 0.5–5.1 | 0.0–23.5 | 0.0–11.3 | 0.0–11.3 | 0.3–1.0 | 0.3–24.1 | 2.6–10.5 | 0.3–24.1 |
Age at diagnosis | |||||||||||
Median | 6.6 | 7.7 | 6.1 | 7.0 | 6.2 | 2.8 | 4.4 | 1.9 | 10.3 | 8.3 | 10.3 |
Mean ± SD | 8.3 ± 7.2 | 7.3 ± 2.1 | 8.6 ± 8.1 | 8.5 ± 7.3 | 8.0 ± 7.3 | 3.8 ± 3.0 | 5.0 ± 3.6 | 2.1 ± 0.6 | 11.3 ± 7.0 | 8.3 ± 3.1 | 11.7 ± 7.3 |
Range | 0.0–27.2 | 4.5–9.6 | 0.0–27.2 | 1.3–27.2 | 0.0–25.2 | 1.3–9.8 | 1.3–9.8 | 1.6–2.8 | 2.6–24.1 | 6.2–10.5 | 2.6–24.1 |
Age treatment started | |||||||||||
Median | 17.0 | 8.4 | 21.5 | 16.1 | 21.0 | 3.2 | 5.1 | 2.8 | |||
Mean ± SD | 18.9 ± 9.7 | 9.2 ± 3.3 | 21.8 ± 9.1 | 19.8 ± 10.0 | 17.9 ± 9.7 | 4.0 ± 2.4 | 4.9 ± 3.0 | 2.8 ± 0.8 | |||
Range | 5.3–37.5 | 5.3–14.8 | 5.7–37.5 | 7.7–37.5 | 5.3–34.8 | 1.6–7.8 | 1.6–7.8 | 2.0–3.6 | |||
Treatment length | |||||||||||
Median | 5.3 | 2.0 | 5.9 | 1.9 | 10.2 | 10.4 | 7.6 | 17.8 | |||
Mean ± SD | 6.0 ± 4.4 | 3.6 ± 3.8 | 6.7 ± 4.4 | 2.2 ± 1.4 | 9.9 ± 2.5 | 12.0 ± 6.5 | 7.5 ± 3.1 | 17.9 ± 4.2 | |||
Range | 0.3–12.5 | 0.5–10.3 | 0.3–12.5 | 0.3–4.7 | 5.8–12.5 | 4.3–22.2 | 4.3–10.4 | 13.8–22.2 |