Table. Patient Enrollment and Demographicsa.
| Characteristic | Overall cohort | ECO cohort | LCO cohort |
|---|---|---|---|
| Individuals with NPC1, No. | 50 | 30 | 20 |
| Sex, No. (%) | |||
| Male | 24 (48) | 14 (47) | 10 (50) |
| Female | 26 (52) | 16 (53) | 10 (50) |
| Age at neurologic disease onset, median (IQR), y | 3.5 (2.0-7.7) | 2.0 (1.5-3.0) | 8.0 (6.5-11.0) |
| Age at baseline evaluation, median (IQR), y | 9.4 (3.4-16.4) | 4.4 (2.4-10.0) | 16.6 (11.6-21.6) |
| Individuals taking miglustat, No. (%) | |||
| Baseline | 21 (42) | 11 (37) | 10 (50) |
| Follow-up | 36 (72) | 21 (70) | 15 (75) |
| Duration of miglustat treatment, median (IQR), y | 3.1 (1.2-5.4) | 3.2 (1.2-5.5) | 2.9 (1.8-5.2) |
| Evaluations of individuals with seizures, No. (%) | 69 (38) | 51 (43) | 18 (28) |
| Evaluations of individuals taking miglustat, No. (%) | 109 (60) | 66 (56) | 43 (67) |
| Total No. of evaluations | 182 | 118 | 64 |
| Duration of follow-up, median (IQR), y | 3.0 (1.1-4.4) | 3.3 (1.1-4.4) | 2.8 (1.1-4.5) |
Abbreviations: ECO, early childhood onset; IQR, interquartile range; LCO, late childhood onset; NPC1, Niemann-Pick disease, type C1.
a
Neurologic disease presented in individuals younger than 15 years in the overall cohort, younger than 6 years in the ECO cohort, and 6 or older and younger than 15 years in the LCO cohort.