Patient demographics and disease characteristics stratified by Loes score at the time of HCT
| Characteristic . | Loes score < 10 . | Loes score ≥ 10 . | P . |
|---|---|---|---|
| Age at diagnosis, y | < .01 | ||
| Median (range) | 5.2 (0-14.3) | 8.4 (2.5-22.3) | |
| Time from ALD diagnosis*to HCT, y | < .01 | ||
| Median (range) | 1.4 (0-10.1) | 0.2 (0-10.3) | |
| Cerebral disease at HCT | .66 | ||
| Predominant frontal | 3 (10%) | 5 (17%) | |
| Predominant parieto-occipital | 25 (83%) | 22 (73%) | |
| Mixed | 2 (7%) | 3 (10%) | |
| Cerebral disease at diagnosis* | .06 | ||
| Yes | 16 (53%) | 21 (70%) | |
| No | 12 (40%) | 5 (17%) | |
| Unknown | 2 (7%) | 4 (13%) | |
| NFS at HCT | < .01 | ||
| 0 | 20 (67%) | 3 (10%) | |
| 1 | 9 (30%) | 8 (27%) | |
| ≥ 2 | 1 (3%) | 19 (63%) | |
| Neuropsychometric indices, median (range) | |||
| Verbal IQ at HCT | 89 (50-121) | 88 (59-117) | .33 |
| Performance IQ at HCT | 96 (61-131) | 77 (45-100) | < .01 |
| Full Scale IQ at HCT | 91 (45-124) | 75 (51-103) | < .01 |
| Treatment with NAC | .30 | ||
| Yes | 15 (50%) | 19 (63%) | |
| No | 15 (50%) | 11 (37%) |
| Characteristic . | Loes score < 10 . | Loes score ≥ 10 . | P . |
|---|---|---|---|
| Age at diagnosis, y | < .01 | ||
| Median (range) | 5.2 (0-14.3) | 8.4 (2.5-22.3) | |
| Time from ALD diagnosis*to HCT, y | < .01 | ||
| Median (range) | 1.4 (0-10.1) | 0.2 (0-10.3) | |
| Cerebral disease at HCT | .66 | ||
| Predominant frontal | 3 (10%) | 5 (17%) | |
| Predominant parieto-occipital | 25 (83%) | 22 (73%) | |
| Mixed | 2 (7%) | 3 (10%) | |
| Cerebral disease at diagnosis* | .06 | ||
| Yes | 16 (53%) | 21 (70%) | |
| No | 12 (40%) | 5 (17%) | |
| Unknown | 2 (7%) | 4 (13%) | |
| NFS at HCT | < .01 | ||
| 0 | 20 (67%) | 3 (10%) | |
| 1 | 9 (30%) | 8 (27%) | |
| ≥ 2 | 1 (3%) | 19 (63%) | |
| Neuropsychometric indices, median (range) | |||
| Verbal IQ at HCT | 89 (50-121) | 88 (59-117) | .33 |
| Performance IQ at HCT | 96 (61-131) | 77 (45-100) | < .01 |
| Full Scale IQ at HCT | 91 (45-124) | 75 (51-103) | < .01 |
| Treatment with NAC | .30 | ||
| Yes | 15 (50%) | 19 (63%) | |
| No | 15 (50%) | 11 (37%) |
Diagnosis is defined as the first positive plasma VLCFA profile.