Table 1.
Patient Demographics and Baseline Characteristics*
| Characteristic | Value |
|---|---|
| No. of subjects | 133 |
| Female/male | 61 (46%)/72 (54%) |
| Age, median (range), yr | 5.0 (.2-17.9) |
| Weight, median (range), kg | 15.7 (3-111) |
| Body surface area, median (range), m2 | .68 (.2-2.3) |
| Serum creatinine, median (range), mg/dL | .3 (.14-1.89) |
| Creatinine clearance, median (range), mL/min/m2† | 150 (35-150) |
| Diagnosis | |
| Hematologic malignancies | 59 (44%) |
| Primary immune deficiencies | 18 (14%) |
| Hemoglobinopathies | 8 (6%) |
| Inherited metabolic disorders | 22 (16%) |
| Bone marrow failure | 22 (16%) |
| Epidermolysis bullosa | 4 (4%) |
| Conditioning regimen | |
| Busulfan/fludarabine | 40 (30%) |
| Cyclophosphamide/fludarabine | 45 (34%) |
| Busulfan/fludarabine/clofarabine | 18 (14%) |
| Fludarabine/thiotepa/melphalan | 15 (11%) |
| Other | 15 (11%) |
| Serotherapy | |
| Antithymocyte globulin | 60 (45%) |
| Alemtuzumab | 33 (25%) |
| None | 40 (30%) |
| Donor relation | |
| Related | 38 (29%) |
| Unrelated | 95 (71%) |
| Donor Source | |
| Bone marrow | 62 (47%) |
| Umbilical cord blood | 38 (29%) |
| Peripheral blood stem cells | 33 (24%) |
| Degree of HLA mismatch | |
| Bone marrow or peripheral blood | |
| Fully matched | 44 (46%) |
| 1 Degree mismatch | 36 (38%) |
| ≥ 2 Degrees mismatch | 15 (16%) |
| Umbilical Cord | |
| Fully matched | 11 (29%) |
| 1 Degree mismatch | 21 (55%) |
| ≥ 2 Degrees mismatch | 6 (16%) |
| Fludarabine daily dose | |
| 40 mg/m2 | 55 (41%) |
| 12.5-35 mg/m2 | 40 (30%) |
| .9-1.33 mg/kg | 38 (29%) |
Data presented are n (%) unless otherwise indicated.
*
Laboratory data was collected just prior to the first dose of fludarabine.
†
Creatinine clearance was estimated in children using the Schwartz method and in young adults greater than 17 years of age by the Cockcroft-Gault equation using ideal body weight.