Study cohort demographic and disease characteristics
| Characteristic . | Patients with TMA (n = 34) . | Patients without TMA (n = 43) . | P . | ||
|---|---|---|---|---|---|
| No. . | % . | No. . | % . | ||
| Age, y (range) | 9.19 (4.0-16.0) | 7.1 (1.3-16.0) | .35 | ||
| Male | 23 | 67.6 | 27 | 62.7 | .42 |
| Diagnosis | .02 | ||||
| Bone marrow failure | 12 | 35.3 | 4 | 9.3 | |
| Immunodeficiency | 14 | 41.2 | 18 | 41.9 | |
| Malignancy | 7 | 20.6 | 19 | 44.2 | |
| Genetic/metabolic | 1 | 2.9 | 2 | 4.7 | |
| Donor type | 1 | ||||
| Related | 9 | 26.5 | 12 | 27.9 | |
| Unrelated | 25 | 73.5 | 31 | 72.1 | |
| Race | .04 | ||||
| White | 24 | 70.6 | 39 | 9.7 | |
| Nonwhite | 10 | 29.4 | 4 | 9.3 | |
| Stem cell source | .01 | ||||
| Bone marrow | 23 | 67.7 | 38 | 88.4 | |
| PBSCs | 8 | 23.5 | 1 | 2.3 | |
| Cord blood | 3 | 8.8 | 4 | 9.3 | |
| HLA match | |||||
| Bone marrow | .53 | ||||
| 8/8 | 17 | 2373.9 | 31 | 3881.6 | |
| 7/8 | 6 | 2326.1 | 7 | 3818.4 | |
| PBSCs | 1 | ||||
| 8/8 | 5 | 862.5 | 1 | 1100 | |
| 7/8 | 3 | 837.5 | 0 | 10 | |
| Cord blood | .43 | ||||
| 6/6 | 1 | 333.3 | 0 | 40 | |
| 5/6 | 2 | 366.7 | 4 | 4100 | |
| Conditioning regimen | .65 | ||||
| Myeloablative | 17 | 50 | 24 | 55.8 | |
| Reduced intensity | 17 | 50 | 19 | 44.2 | |
| GVHD prophylaxis | |||||
| CSA + steroids | 21 | 61.8 | 22 | 51.2 | .37 |
| CSA + methotrexate | 4 | 11.8 | 16 | 37.2 | .02 |
| CSA + mycophenolate | 1 | 2.9 | 2 | 4.7 | 1 |
| CSA + T-cell depletion | 6 | 17.6 | 1 | 2.3 | .13 |
| Mycophenolate + steroids | 1 | 2.9 | 1 | 2.3 | 1 |
| Tacrolimus or sirolimus | 1 | 2.9 | 1 | 2.3 | 1 |
| Characteristic . | Patients with TMA (n = 34) . | Patients without TMA (n = 43) . | P . | ||
|---|---|---|---|---|---|
| No. . | % . | No. . | % . | ||
| Age, y (range) | 9.19 (4.0-16.0) | 7.1 (1.3-16.0) | .35 | ||
| Male | 23 | 67.6 | 27 | 62.7 | .42 |
| Diagnosis | .02 | ||||
| Bone marrow failure | 12 | 35.3 | 4 | 9.3 | |
| Immunodeficiency | 14 | 41.2 | 18 | 41.9 | |
| Malignancy | 7 | 20.6 | 19 | 44.2 | |
| Genetic/metabolic | 1 | 2.9 | 2 | 4.7 | |
| Donor type | 1 | ||||
| Related | 9 | 26.5 | 12 | 27.9 | |
| Unrelated | 25 | 73.5 | 31 | 72.1 | |
| Race | .04 | ||||
| White | 24 | 70.6 | 39 | 9.7 | |
| Nonwhite | 10 | 29.4 | 4 | 9.3 | |
| Stem cell source | .01 | ||||
| Bone marrow | 23 | 67.7 | 38 | 88.4 | |
| PBSCs | 8 | 23.5 | 1 | 2.3 | |
| Cord blood | 3 | 8.8 | 4 | 9.3 | |
| HLA match | |||||
| Bone marrow | .53 | ||||
| 8/8 | 17 | 2373.9 | 31 | 3881.6 | |
| 7/8 | 6 | 2326.1 | 7 | 3818.4 | |
| PBSCs | 1 | ||||
| 8/8 | 5 | 862.5 | 1 | 1100 | |
| 7/8 | 3 | 837.5 | 0 | 10 | |
| Cord blood | .43 | ||||
| 6/6 | 1 | 333.3 | 0 | 40 | |
| 5/6 | 2 | 366.7 | 4 | 4100 | |
| Conditioning regimen | .65 | ||||
| Myeloablative | 17 | 50 | 24 | 55.8 | |
| Reduced intensity | 17 | 50 | 19 | 44.2 | |
| GVHD prophylaxis | |||||
| CSA + steroids | 21 | 61.8 | 22 | 51.2 | .37 |
| CSA + methotrexate | 4 | 11.8 | 16 | 37.2 | .02 |
| CSA + mycophenolate | 1 | 2.9 | 2 | 4.7 | 1 |
| CSA + T-cell depletion | 6 | 17.6 | 1 | 2.3 | .13 |
| Mycophenolate + steroids | 1 | 2.9 | 1 | 2.3 | 1 |
| Tacrolimus or sirolimus | 1 | 2.9 | 1 | 2.3 | 1 |
PBSC, peripheral blood stem cell.