Patient demographics and HCT characteristics
| Factor . | No. (%) . |
|---|---|
| Total cALD cohort | 60 (100) |
| Reason for diagnosis | |
| Family history | 17 (28) |
| Signs/symptoms | 37 (62) |
| Unknown | 6 (10) |
| Months from diagnosis*to HCT | |
| Median (range), (IQR) | 5.1 (0.7-123), (2.7-25.4) |
| Location of cerebral disease at HCT | |
| Predominant frontal | 8 (13) |
| Predominant parieto-occipital | 49 (82) |
| Mixed | 3 (5) |
| Loes score at HCT | |
| < 10 | 30 (50) |
| ≥ 10 | 30 (50) |
| NFS at HCT | |
| 0 | 23 (38) |
| 1 | 17 (29) |
| ≥ 2 | 20 (33) |
| Adrenal insufficiency before HCT | |
| Yes | 43 (72) |
| No | 10 (17) |
| Unknown | 7 (12) |
| Age at HCT, y | |
| Median (range), (IQR) | 8.7 (4-23.3), (7-10.1) |
| Year of HCT | |
| 2000-2005 | 28 (47) |
| 2006-2009 | 32 (53) |
| Donor type | |
| Related marrow | 18 (30) |
| Unrelated marrow | 10 (17) |
| Unrelated UCB (single) | 12 (20) |
| Unrelated UCB (double) | 20 (33) |
| HLA compatibility | |
| Matched | 27 (45) |
| Mismatched | 33 (55) |
| Preparative regimen | |
| Bu/Cy-based | 28 (46) |
| Cy/TBI-based | 16 (27) |
| RIC | 16 (27) |
| Peri-HCT NAC therapy | |
| Yes | 34 (57) |
| No | 26 (43) |
| Factor . | No. (%) . |
|---|---|
| Total cALD cohort | 60 (100) |
| Reason for diagnosis | |
| Family history | 17 (28) |
| Signs/symptoms | 37 (62) |
| Unknown | 6 (10) |
| Months from diagnosis*to HCT | |
| Median (range), (IQR) | 5.1 (0.7-123), (2.7-25.4) |
| Location of cerebral disease at HCT | |
| Predominant frontal | 8 (13) |
| Predominant parieto-occipital | 49 (82) |
| Mixed | 3 (5) |
| Loes score at HCT | |
| < 10 | 30 (50) |
| ≥ 10 | 30 (50) |
| NFS at HCT | |
| 0 | 23 (38) |
| 1 | 17 (29) |
| ≥ 2 | 20 (33) |
| Adrenal insufficiency before HCT | |
| Yes | 43 (72) |
| No | 10 (17) |
| Unknown | 7 (12) |
| Age at HCT, y | |
| Median (range), (IQR) | 8.7 (4-23.3), (7-10.1) |
| Year of HCT | |
| 2000-2005 | 28 (47) |
| 2006-2009 | 32 (53) |
| Donor type | |
| Related marrow | 18 (30) |
| Unrelated marrow | 10 (17) |
| Unrelated UCB (single) | 12 (20) |
| Unrelated UCB (double) | 20 (33) |
| HLA compatibility | |
| Matched | 27 (45) |
| Mismatched | 33 (55) |
| Preparative regimen | |
| Bu/Cy-based | 28 (46) |
| Cy/TBI-based | 16 (27) |
| RIC | 16 (27) |
| Peri-HCT NAC therapy | |
| Yes | 34 (57) |
| No | 26 (43) |
Male-related marrow donors (n = 11) were excluded of disease by plasma VLCFA profile testing; female-related marrow donors (n = 7) were tested for carrier status by ABCD1 gene analysis: 4 indicates wild-type; 2, heterozygote carrier; and 1, unknown status.
IQR indicates interquartile range; and UCB, umbilical cord blood.
Diagnosis is defined as the first positive plasma VLCFA profile.