Characteristics of 94 patients with cerebral X-adrenoleukodystrophy (X-ALD) who underwent hematopoietic cell transplant (HC transplant)
. | Related donor (n = 42) . | Unrelated donor (n = 52) . | Total (n = 94) . |
|---|---|---|---|
| Year of HCT, no. (%) | |||
| Before 1990 | 5 (12) | 0 (0) | 5 (5) |
| 1991 to 1995 | 24 (56) | 20 (38) | 44 (46) |
| 1996 to 1999 | 13 (31) | 32 (62) | 45 (48) |
| Median age at HCT, y (range) | 9.1 (5.2-15.6) | 8.9 (4.9-18.6) | 9.0 (4.9-18.6) |
| Reason for diagnosis, no. (%)* | |||
| Family history | 11 (29) | 17 (35) | 28 (33) |
| X-ALD signs and symptoms | 27 (71) | 31 (65) | 58 (67) |
| Unknown | 4 | 4 | 8 |
| Stem cell source and HLA matching | |||
| Marrow/UCB | 42/0 | 40/12 | 82/12 |
| Matched | 33/0 | 27/4 | 60/4 |
| Mismatched | 9/0 | 13/8 | 22/8 |
| Preparative regimen, no. (%) | |||
| Chemotherapy only | 31 (74) | 17 (33) | 48 (51) |
| Bu/Cy | 30 (97) | 17 (100) | 47 (98) |
| Bu only | 1 (3) | 0 | 1 (2) |
| Chemotherapy and radiation | 11 (26) | 35 (67) | 46 (49) |
| Cy/TBI, brain-sparing at 1400 cGy | 5 (45) | 13 (37) | 18 (39) |
| Bu/Cy/TBI at 300-1200 cGy | 1 (9) | 9 (26) | 10 (22) |
| Cy/TBI at 1200-1400 cGy | 1 (9) | 8 (23) | 9 (20) |
| Bu/Cy/TLI at 150-750 cGy | 2 (18) | 1 (3) | 3 (7) |
| Cy/TAI at 800 cGy | 1 (9) | 1 (3) | 2 (4) |
| VP-16/Ara-C/Cy/TBI at 1200 cGy | 1 (9) | 1 (3) | 2 (4) |
| Other | 0 | 2 (6) | 2 (4) |
| GVHD prophylaxis | |||
| CSA/MP, no. (%) | 24 (57) | 29 (56) | 53 (56) |
| No. with/no. without ATG | 3/21 | 12/17 | 15/38 |
| CSA/MTX, no. (%) | 9 (21) | 8 (15) | 17 (18) |
| No. with/no. without ATG | 2/7 | 6/2 | 8/9 |
| Other, no. (%) | 9 (21) | 15 (29) | 24 (26) |
| Graft manipulation by elutriation, no. yes/no. no | 3/39 | 15/37 | 18/76 |
. | Related donor (n = 42) . | Unrelated donor (n = 52) . | Total (n = 94) . |
|---|---|---|---|
| Year of HCT, no. (%) | |||
| Before 1990 | 5 (12) | 0 (0) | 5 (5) |
| 1991 to 1995 | 24 (56) | 20 (38) | 44 (46) |
| 1996 to 1999 | 13 (31) | 32 (62) | 45 (48) |
| Median age at HCT, y (range) | 9.1 (5.2-15.6) | 8.9 (4.9-18.6) | 9.0 (4.9-18.6) |
| Reason for diagnosis, no. (%)* | |||
| Family history | 11 (29) | 17 (35) | 28 (33) |
| X-ALD signs and symptoms | 27 (71) | 31 (65) | 58 (67) |
| Unknown | 4 | 4 | 8 |
| Stem cell source and HLA matching | |||
| Marrow/UCB | 42/0 | 40/12 | 82/12 |
| Matched | 33/0 | 27/4 | 60/4 |
| Mismatched | 9/0 | 13/8 | 22/8 |
| Preparative regimen, no. (%) | |||
| Chemotherapy only | 31 (74) | 17 (33) | 48 (51) |
| Bu/Cy | 30 (97) | 17 (100) | 47 (98) |
| Bu only | 1 (3) | 0 | 1 (2) |
| Chemotherapy and radiation | 11 (26) | 35 (67) | 46 (49) |
| Cy/TBI, brain-sparing at 1400 cGy | 5 (45) | 13 (37) | 18 (39) |
| Bu/Cy/TBI at 300-1200 cGy | 1 (9) | 9 (26) | 10 (22) |
| Cy/TBI at 1200-1400 cGy | 1 (9) | 8 (23) | 9 (20) |
| Bu/Cy/TLI at 150-750 cGy | 2 (18) | 1 (3) | 3 (7) |
| Cy/TAI at 800 cGy | 1 (9) | 1 (3) | 2 (4) |
| VP-16/Ara-C/Cy/TBI at 1200 cGy | 1 (9) | 1 (3) | 2 (4) |
| Other | 0 | 2 (6) | 2 (4) |
| GVHD prophylaxis | |||
| CSA/MP, no. (%) | 24 (57) | 29 (56) | 53 (56) |
| No. with/no. without ATG | 3/21 | 12/17 | 15/38 |
| CSA/MTX, no. (%) | 9 (21) | 8 (15) | 17 (18) |
| No. with/no. without ATG | 2/7 | 6/2 | 8/9 |
| Other, no. (%) | 9 (21) | 15 (29) | 24 (26) |
| Graft manipulation by elutriation, no. yes/no. no | 3/39 | 15/37 | 18/76 |
UCB indicates umbilical cord blood; Bu, busulfan; Cy, cyclophosphamide; TBI, total body irradiation; TLI, total lymphoid irradiation; TAI, total abdominal irradiation; VP-16, etoposide; Ara-C, cytosine arabinoside; CSA, cyclosporin; MP, methylprednisolone; and ATG, antithymocyte globulin.
Percentages were calculated only for patients with a known reason