Table 4. Patient Characteristics and Outcome by Different Subgroups.
| Variable | All Patients, No. (%) (N = 36) | Patients, No. | |||
|---|---|---|---|---|---|
| Favorable Neuroimaging | Unfavorable Neuroimaging | ||||
| Bone Marrow (n = 11), Group Ia | Peripheral Stem Cells or Cord Blood (n = 7), Group Ib | With Demyelination of Cerebellum or Basal Ganglia (n = 4), Group IIa | Other MRI (n = 14), Group IIb | ||
| Neurological status at HSCT | |||||
| Presymptomatic | 18 (50) | 10 | 7 | 0 | 1 |
| NFS ≤1a | 29 (81) | 11 | 7 | 2 | 9 |
| ALD-DRS = 0a | 20 (56) | 10 | 7 | 1 | 2 |
| Neuroimaging before HSCT | |||||
| Loes score ≥9a | 8 (22) | 0 | 0 | 1 | 7 |
| Parieto-occipital pattern | 22 (61) | 9 | 7 | 0 | 6 |
| Projection fibers involved | 21 (58) | 4 | 1 | 3 | 13 |
| Transplant characteristics | |||||
| Transplant before 2004 | 21 (58) | 4 | 4 | 4 | 9 |
| Related donor | 9 (25) | 5 | 0 | 0 | 4 |
| Bone marrow | 26 (72) | 11 | 0 | 3 | 12 |
| Engraftment before day 14 after HSCT | 16 (44) | 4 | 4 | 1 | 7 |
| Donor chimerism >95% | 31 (89)b | 9b | 7 | 3 | 12 |
| Infection grade >2 | 14 (39) | 2 | 3 | 2 | 7 |
| Acute GVHD grade ≥2 | 9 (25) | 0 | 2 | 2 | 5 |
| Outcome | |||||
| Transplant-related deaths | 3 (8) | 0 | 2 | 0 | 1 |
| Disease-related deaths | 6 (17) | 0 | 0 | 4 | 2 |
| Major functional disabilities | 10 (28) | 0 | 0 | 4 | 6 |
| Event-free survivors | 14 (39) | 11 | 3 | 0 | 0 |
| Event-free survival time, median (IQR), moc | 26 (9-60) | 104 (45-121) | 38 (17-132) | 6 (2.5-9.2) | 10 (4.8-22) |
Abbreviations: ALD-DRS, adrenoleukodystrophy disability rating score; GVHD, graft-vs-host disease; HSCT, hematopoietic stem cell transplantation; IQR, interquartile range; MRI, magnetic resonance imaging; NFS, neurological function score.
a
ALD-DRS range, 0-4 points; NFS range, 0-25 points (NFS ≤1: score with maximum 1 deficit); and Loes MRI score range, 0-34 points. For all scores: 0 indicates no abnormalities, and higher numbers indicate worsening status.
b
Donor chimerism: determined in only of 35 patients (1 patient from group Ia was missing).
c
Kaplan-Meier estimates.