Patient characteristics by center
| Factors . | Center . | |||||
|---|---|---|---|---|---|---|
| CCHMC . | DFCI/CHB . | UMACH . | TXCCC . | n . | P . | |
| Total | 86 | 48 | 53 | 65 | 252 | |
| Diagnosis | < .01* | |||||
| Aplastic anemia | 6 (7%) | 5 (10%) | 5 (9%) | 6 (9%) | 22 | |
| Epidermolysis bullosa | 0 | 0 | 2 (4%) | 0 | 2 | |
| Fanconi anemia | 12 (14%) | 0 | 5 (9%) | 1 (2%) | 18 | |
| Immune deficiency | 45 (52%) | 5 (10%) | 4 (8%) | 9 (14%) | 63 | |
| Storage disorder | 2 (2%) | 0 | 20 (38%) | 2 (3%) | 24 | |
| Leukemia | 21 (24%) | 35 (73%) | 16 (30%) | 38 (58%) | 110 | |
| MFS/MPS | 0 | 0 | 0 | 1 (2%) | 1 | |
| Solid tumor | 0 | 0 | 0 | 3 (5%) | 3 | |
| Benign hematologic disorders | 0 | 3 (6%) | 1 (2%) | 5 (8%) | 9 | |
| Age, y | .48 | |||||
| 0-1 | 22 (26%) | 9 (19%) | 14 (26%) | 11 (17%) | 56 | |
| 2-9 | 41 (48%) | 21 (44%) | 27 (51%) | 28 (43%) | 117 | |
| 10-17 | 19 (22%) | 13 (27%) | 11 (21%) | 22 (34%) | 65 | |
| 18-20 | 4 (5%) | 5 (10%) | 1 (2%) | 4 (6%) | 14 | |
| Median (range) | 4 (0.2-19) | 7 (0.1-19) | 6 (0.1-20) | 8 (0.3-19) | 6 (0.1-20) | |
| Donor type | < .01* | |||||
| Matched RD | 14 (16%) | 14 (29%) | 20 (38%) | 26 (40%) | 74 | |
| Mismatched RD | 3 (3%) | 0 | 3 (6%) | 15 (23%) | 21 | |
| Matched URD BM/PBSC | 36 (42%) | 14 (29%) | 4 (8%) | 21 (32%) | 75 | |
| Mismatched URD BM/PBSC | 20 (23%) | 12 (25%) | 1 (2%) | 1 (2%) | 34 | |
| UCB | 13 (15%) | 8 (17%) | 25 (47%) | 2 (3%) | 48 | |
| Conditioning | < .01* | |||||
| RIC/NMA | 36 (42%) | 0 | 9 (17%) | 16 (25%) | 61 | |
| MA | 50 (58%) | 48 (100%) | 44 (83%) | 47 (72%) | 189 | |
| None | 0 | 0 | 0 | 2 (3%) | 2 | |
| HCT-CI | < .01* | |||||
| 0 | 59 (69%) | 28 (58%) | 34 (64%) | 18 (28%) | 139 | |
| 1 | 9 (10%) | 3 (6%) | 11 (21%) | 10 (15%) | 33 | |
| 2 | 2 (2%) | 7 (15%) | 2 (4%) | 8 (12%) | 19 | |
| 3+ | 16 (19%) | 10 (21%) | 6 (11%) | 29 (45%) | 61 | |
| Median (range) | 0 (0-6) | 0 (0-7) | 0 (0-5) | 2 (0-9) | 0 (0-9) | |
| Median days of follow-up (range) | 349 (110-624) | 321 (130-620) | 319 (117-551) | 366 (116-606) | 343 (110-624) | .38 |
| Factors . | Center . | |||||
|---|---|---|---|---|---|---|
| CCHMC . | DFCI/CHB . | UMACH . | TXCCC . | n . | P . | |
| Total | 86 | 48 | 53 | 65 | 252 | |
| Diagnosis | < .01* | |||||
| Aplastic anemia | 6 (7%) | 5 (10%) | 5 (9%) | 6 (9%) | 22 | |
| Epidermolysis bullosa | 0 | 0 | 2 (4%) | 0 | 2 | |
| Fanconi anemia | 12 (14%) | 0 | 5 (9%) | 1 (2%) | 18 | |
| Immune deficiency | 45 (52%) | 5 (10%) | 4 (8%) | 9 (14%) | 63 | |
| Storage disorder | 2 (2%) | 0 | 20 (38%) | 2 (3%) | 24 | |
| Leukemia | 21 (24%) | 35 (73%) | 16 (30%) | 38 (58%) | 110 | |
| MFS/MPS | 0 | 0 | 0 | 1 (2%) | 1 | |
| Solid tumor | 0 | 0 | 0 | 3 (5%) | 3 | |
| Benign hematologic disorders | 0 | 3 (6%) | 1 (2%) | 5 (8%) | 9 | |
| Age, y | .48 | |||||
| 0-1 | 22 (26%) | 9 (19%) | 14 (26%) | 11 (17%) | 56 | |
| 2-9 | 41 (48%) | 21 (44%) | 27 (51%) | 28 (43%) | 117 | |
| 10-17 | 19 (22%) | 13 (27%) | 11 (21%) | 22 (34%) | 65 | |
| 18-20 | 4 (5%) | 5 (10%) | 1 (2%) | 4 (6%) | 14 | |
| Median (range) | 4 (0.2-19) | 7 (0.1-19) | 6 (0.1-20) | 8 (0.3-19) | 6 (0.1-20) | |
| Donor type | < .01* | |||||
| Matched RD | 14 (16%) | 14 (29%) | 20 (38%) | 26 (40%) | 74 | |
| Mismatched RD | 3 (3%) | 0 | 3 (6%) | 15 (23%) | 21 | |
| Matched URD BM/PBSC | 36 (42%) | 14 (29%) | 4 (8%) | 21 (32%) | 75 | |
| Mismatched URD BM/PBSC | 20 (23%) | 12 (25%) | 1 (2%) | 1 (2%) | 34 | |
| UCB | 13 (15%) | 8 (17%) | 25 (47%) | 2 (3%) | 48 | |
| Conditioning | < .01* | |||||
| RIC/NMA | 36 (42%) | 0 | 9 (17%) | 16 (25%) | 61 | |
| MA | 50 (58%) | 48 (100%) | 44 (83%) | 47 (72%) | 189 | |
| None | 0 | 0 | 0 | 2 (3%) | 2 | |
| HCT-CI | < .01* | |||||
| 0 | 59 (69%) | 28 (58%) | 34 (64%) | 18 (28%) | 139 | |
| 1 | 9 (10%) | 3 (6%) | 11 (21%) | 10 (15%) | 33 | |
| 2 | 2 (2%) | 7 (15%) | 2 (4%) | 8 (12%) | 19 | |
| 3+ | 16 (19%) | 10 (21%) | 6 (11%) | 29 (45%) | 61 | |
| Median (range) | 0 (0-6) | 0 (0-7) | 0 (0-5) | 2 (0-9) | 0 (0-9) | |
| Median days of follow-up (range) | 349 (110-624) | 321 (130-620) | 319 (117-551) | 366 (116-606) | 343 (110-624) | .38 |
CCHMC indicates Cincinnati Children's Hospital Medical Center; DFCI/CHB, Dana-Farber Cancer Institute/Children's Hospital Boston; UMACH, University of Minnesota Amplatz Children's Hospital; TXCCC, Texas Children's Cancer Center; MFS, myelofibrosis syndrome; MPS, myeloproliferative syndrome; RD, related donor; URD, unrelated donor; BM, bone marrow; PBSC, peripheral blood stem cell; UCB, umbilical cord blood; RIC, reduced-intensity conditioning; and NMA, nonmyeloablative.