Table 1.
Patients’ characteristics
| Characteristics | LEUKOFRANCE CCALD cohort (N = 52) | |
|---|---|---|
| Age (y) | median (Q1-Q3) | 7.0 (6.0–9.0) |
| min-max | 0.5–13.0 | |
| Age classes (y) | ||
| < 3 years old | N (%) | 2 (3.8) |
| [3–5[ years old | 2 (3.8) | |
| [5–8[ years old | 27 (51.9) | |
| [8–12[ years old | 17 (32.7) | |
| [12–15[ years old | 4 (7.7) | |
| [15–18[ years old | 0 (0.0) | |
| ALD family history | ||
| yes | N (%) | 15 (29.4) |
| no | 36 (70.6) | |
| Incident cases | ||
| 2009 | N | 2 |
| 2010 | 3 | |
| 2011 | 5 | |
| 2012 | 2 | |
| 2013 | 4 | |
| 2014 | 2 | |
| 2015 | 6 | |
| 2016 | 3 | |
| 2017 | 4 | |
| 2018 | 4 | |
| 10-year study period | N (%) | 35 (67.3) |
| Prevalent cases | N (%) | 17 (32.7) |
| Inclusion status | ||
| Familial screen cases | N (%) | 14 (26.9) |
| Index cases | N (%) | 38 (73.1) |
| Hematopoietic stem cell transplant (HSCT) status | ||
| Patients with allogenic HSCT | N (%) | 19 (36.5) |
| Patients without allogenic HSCT | N (%) | 29 (55.8) |
| Patients with gene and auto HSCT therapy | N (%) | 3 (5.8) |
| Missing value | N (%) | 1 (1.9) |
ALD: adrenoleukodystrophy; CCALD: childhood cerebral ALD