Table 2. Analysis of Transplant Outcomes.
| Variable | No. (%) | Survival | ||
|---|---|---|---|---|
| Overall | MFD-Freea | Event-Freea | ||
| All patients [events] | 36 (100) | [8] | [13] | [22] |
| Kaplan-Meier Estimatesb | ||||
| Survival times, mo | ||||
| Median (IQR) | 36 (100) | 108 (40-156) | 69 (23-131) | 26 (9-60) |
| 10-y Survival probability, % | ||||
| Mean (95% CI) | 36 (100) | 77 (64-92) | 64 (50-82) | 39 (26-59) |
| Hazard Ratios (95% CI)c | ||||
| Neurological status at HSCT | ||||
| Presymptomatic | 18 (50) | 0.31 (0.06-1.5) | 0.13 (0.03-0.57) | 0.12 (0.04-0.33) |
| NFS ≤1d | 29 (81) | 0.29 (0.07-1.2) | 0.11 (0.04-0.34) | 0.11 (0.04-0.31) |
| ALD-DRS = 0d | 20 (56) | 0.43 (0.10-1.8) | 0.15 (0.04-0.56) | 0.17 (0.07-0.42) |
| Neuroimaging before HSCT | ||||
| Loes score ≥9d | 8 (22) | 0.48 (0.06-4.0) | 3.0 (1.0-9.1) | 4.4 (1.8-10.9) |
| Cerebellar patterne | 4 (11) | 12.1 (3.0-49.0) | 6.5 (2.0-21.5) | 6.2 (1.9-20.9) |
| Projection fibers involved | 21 (58) | 1.3 (0.3-5.3) | 2.1 (0.6-6.7) | 4.0 (1.5-11.1) |
| Unfavorable MRI | 18 (50) | 3.2 (0.7-16.1) | 8.0 (1.8-36.1) | 16.7 (4.7-59.6) |
| Transplant characteristics | ||||
| Related donor | 9 (25) | NAf | 0.20 (0.03-1.5) | 0.50 (0.17-1.5) |
| Bone marrow | 26 (72) | 0.67 (0.16-2.8) | 0.93 (0.29-3.0) | 0.84 (0.34-2.1) |
| Engraftment before day 14 after HSCT | 16 (44) | 1.2 (0.31-5.0) | 1.2 (0.39-3.5) | 1.4 (0.62-3.3) |
| Infection grade >2 | 14 (39) | 5.5 (1.1-27.4) | 7.0 (1.9-25.9) | 3.0 (1.3-7.0) |
| Acute GVHD grade ≥2 | 9 (25) | 6.0 (1.4-25.3) | 3.2 (1.1-9.7) | 3.0 (1.3-7.1) |
| Extensive chronic GVHD | 8 (22) | 7.4 (1.8-31.2) | 4.1 (1.3-12.3) | 3.3 (1.3-8.5) |
Abbreviations: ALD-DRS, adrenoleukodystrophy disability rating score; GVHD, graft-vs-host disease; HSCT, hematopoietic stem cell transplantation; IQR, interquartile range; MFD, major functional disability; MRI, magnetic resonance imaging; NA, not applicable; NFS, neurological function score.
MFD-free is survival without major functional disabilities. Event-free is survival without gain in ALD-DRS.
To allow calculation of 95% CIs, maximum follow-up was limited to 180 months after HSCT (n = 4).
Cox proportional hazards regression of covariates was used for respective outcomes.
ALD-DRS range, 0-4 points; NFS range, 0-25 points (NFS ≤1: score with maximum 1 deficit); and Loes in this MRI score range, 0-34 points. For all scores: 0 indicates no abnormalities, and higher numbers indicate worsening status.
Cerebellar pattern refers to demyelinating lesions in the cerebellum and/or basal ganglia.
Hazard ratio and 95% CI cannot be calculated due to 100% survival after related bone marrow transplants.