Patient and transplant characteristics
| . | N = 105 (%) . |
|---|---|
| Age at HCT (years) | |
| Median | 4.7 |
| Minimum | 0.3 |
| Maximum | 27.2 |
| Underlying diagnosis | |
| Hemophagocytic lymphohistiocytosis, X-linked lymphoproliferative disease | 54 (51%) |
| Marrow failure (non-Fanconi anemia)* | 13 (12%) |
| Severe combined immune deficiency | 11 (11%) |
| Combined immune deficiency or common variable immune deficiency | 6 (6%) |
| Chronic granulomatous disease | 5 (5%) |
| Metabolic disorder | 4 (4%) |
| Sickle cell disease | 2 (2%) |
| Immunodysregulation, polyendocrinopathy, X-linked syndrome | 2 (2%) |
| Other | 8 (8%) |
| Patient/donor HLA match and relation | |
| Matched related donor | 21 (20%) |
| Matched unrelated donor | 62 (59%) |
| Mismatched donor, 1 or 2 alleles | 22 (21%) |
| GVHD prophylaxis | |
| Calcineurin inhibitor + methylprednisolone | 91 (87%) |
| Calcineurin inhibitor + methylprednisolone + methotrexate | 9 (9%) |
| Other | 5 (5%) |
| Graft source | |
| Bone marrow | 101 (96%) |
| Peripheral blood stem cell | 4 (4%) |
| Total nucleated cell dose (×108/kg) | |
| Median | 7.0 |
| Minimum | 1.2 |
| Maximum | 31.8 |
| Alemtuzumab dosing | |
| Distal alemtuzumab (beginning day −22) | 17 (16%) |
| Intermediate alemtuzumab (beginning day −14) | 69 (66%) |
| Proximal alemtuzumab (beginning day −12 or closer to day 0) | 13 (12%) |
| Unique alemtuzumab (various) | 6 (6%) |
| . | N = 105 (%) . |
|---|---|
| Age at HCT (years) | |
| Median | 4.7 |
| Minimum | 0.3 |
| Maximum | 27.2 |
| Underlying diagnosis | |
| Hemophagocytic lymphohistiocytosis, X-linked lymphoproliferative disease | 54 (51%) |
| Marrow failure (non-Fanconi anemia)* | 13 (12%) |
| Severe combined immune deficiency | 11 (11%) |
| Combined immune deficiency or common variable immune deficiency | 6 (6%) |
| Chronic granulomatous disease | 5 (5%) |
| Metabolic disorder | 4 (4%) |
| Sickle cell disease | 2 (2%) |
| Immunodysregulation, polyendocrinopathy, X-linked syndrome | 2 (2%) |
| Other | 8 (8%) |
| Patient/donor HLA match and relation | |
| Matched related donor | 21 (20%) |
| Matched unrelated donor | 62 (59%) |
| Mismatched donor, 1 or 2 alleles | 22 (21%) |
| GVHD prophylaxis | |
| Calcineurin inhibitor + methylprednisolone | 91 (87%) |
| Calcineurin inhibitor + methylprednisolone + methotrexate | 9 (9%) |
| Other | 5 (5%) |
| Graft source | |
| Bone marrow | 101 (96%) |
| Peripheral blood stem cell | 4 (4%) |
| Total nucleated cell dose (×108/kg) | |
| Median | 7.0 |
| Minimum | 1.2 |
| Maximum | 31.8 |
| Alemtuzumab dosing | |
| Distal alemtuzumab (beginning day −22) | 17 (16%) |
| Intermediate alemtuzumab (beginning day −14) | 69 (66%) |
| Proximal alemtuzumab (beginning day −12 or closer to day 0) | 13 (12%) |
| Unique alemtuzumab (various) | 6 (6%) |
Marrow failure: dyskeratosis congenita, n = 5; aplastic anemia, n = 5; Schwachman-Diamond syndrome, n = 2; congenital thrombocytopenia, n = 1.