Table 1.
Summary of initial patient characteristics.
| Natural history N = 96 | Symptomatic HSCT N = 18 | Asymptomatic HSCT N = 23 | ||||
|---|---|---|---|---|---|---|
| Median | Range | Median | Range | Median | Range | |
| Age at first evaluation (months) | 8.4 | 0.8–47.8 | 6.7 | 4.2–23.0 | 0.8 | 0.1–40.1 |
| Number of evaluations | 2 | 1–9 | 3.5 | 1–10 | 7 | 1–11 |
| Years followed | 0 | 0–10.5 | 1.8 | 0–10.3 | 8.0 | 0–17.9 |
| Age at first symptoms (months) | 4 | 0–12 | 4.5 | 1.5–12 | 1 | 0.3–4.0 |
| Age at diagnosis (months) | 7 | 0–36 | 6.3 | 4–23 | 0 | 0–0.25 |
| Age at HSCT (months) | – | – | 8 | 5.1–23.7 | 1.1 | 0.6–4.9 |
| Initial GALC levels | 0.05 | 0–1.10 | 0.03 | 0–0.12 | 0.07 | 0–0.23 |
| Initial CSF protein levels | 199 | 34–594 | 219 | 83–571 | 191 | 39–528 |
| Initial psychosine levels | 19.7 | 0.2–82.3 | 38.9 | 5.0–54.0 | 6.3 | 0.5–29.1 |
One hundred and thirty‐seven children with infantile‐onset Krabbe disease were included in this study (68 males and 69 females). Of the 137 patients, 96 did not receive HSCT and were considered natural history patients and 41 underwent HSCT, of whom 18 were symptomatic and 23 were asymptomatic at the time of transplantation. Eighty‐seven patients were followed longitudinally with the median number of evaluations as 2 for the natural history patients, 3.5 for the symptomatic HSCT patients, and 7 for the asymptomatic HSCT patients.