Table 4 Demographics and disease characteristics of patients continuously treated with miglustat in the NPC Registry by age at neurological onset category (N = 241)
Patients continuously treated with miglustat | Patients with age at neurological onset dataa | ||||
|---|---|---|---|---|---|
Early-infantile (< 2 years) | Late-infantile (2 to < 6 years) | Juvenile (6 to < 15 years) | Adolescent/adult onset (≥ 15 years) | ||
Age at onset of neurological manifestations | |||||
n (%a) | 224 (100.0) | 21 (9.4) | 66 (29.5) | 81 (36.2) | 56 (25.0) |
Mean (SD), years | 11.2 (10.2) | 0.9 (0.6) | 4.0 (1.3) | 9.9 (2.7) | 25.5 (9.7) |
Median (range), years | 8.1 (0.0–54.8) | 1.0 (0.0–2.0) | 3.9 (2.0–6.0) | 9.6 (6.1–15.0) | 22.1 (15.3–54.8) |
Age at diagnosis | |||||
n (%a) | 171 (100.0) | 17 (10.4) | 53 (32.5) | 56 (34.4) | 37 (22.7) |
Mean (SD), years | 15.0 (11.9) | 4.6 (5.5) | 8.1 (6.9) | 14.8 (6.4) | 30.5 (11.5) |
Median (range), years | 12.7 (0.1–68.8) | 2.5 (0.1–21.4) | 7.3 (0.1–28.2) | 13.7 (2.9–40.2) | 28.1 (14.4–68.8) |
Composite disability score at enrollment | |||||
n (%a) | 221 (100.0) | 19 (8.6) | 60 (27.1) | 76 (34.4) | 53 (24.0) |
Mean (SD) | 0.38 (0.26) | 0.59 (0.35) | 0.34 (0.28) | 0.43 (0.25) | 0.32 (0.16) |
Median (range) | 0.29 (0.00–1.00) | 0.63 (0.00–1.00) | 0.29 (0.00–1.00) | 0.43 (0.00–1.00) | 0.29 (0.00–0.81) |
95% CI of mean | 0.35–0.42 | 0.42–0.76 | 0.27–0.42 | 0.38–0.49 | 0.28–0.37 |
Composite disability score at last follow-up | |||||
n (%a) | 235 (100.0) | 19 (8.1) | 63 (26.8) | 80 (34.0) | 56 (23.8) |
Mean (SD) | 0.48 (0.29) | 0.70 (0.34) | 0.51 (0.32) | 0.49 (0.26) | 0.39 (0.23) |
Median (range) | 0.44 (0.00–1.00) | 0.94 (0.00–1.00) | 0.50 (0.00–1.00) | 0.49 (0.00–1.00) | 0.31 (0.00–0.94) |
95% CI of mean | 0.44–0.51 | 0.54–0.87 | 0.43–0.59 | 0.43–0.55 | 0.33–0.45 |