TABLE I.
Patient and transplant characteristics (N = 81)
| Characteristic | n |
|---|---|
| Sex | |
| Female | 43 |
| Male | 38 |
| Age (y) at HSCT, median (range) | 9.7 (0.7–27.2) |
| Donor type | |
| MSD | 34 |
| MFD | 6 |
| MUD | 32 |
| MMUD | 1 |
| HLA match | |
| 10/10 | 20 |
| 9/10 | 10 |
| 8/10 | 1 |
| 8/8 | 1 |
| 6/6 | 1 |
| MMFD | 6 |
| UCB | 2 |
| Stem cell source | |
| Bone marrow | 63 |
| PBSC | 16 |
| Cord blood | 2 |
| Conditioning | |
| Busulfan-based | 48 |
| Myeloablative | 31 |
| BU/CY | 12 |
| BU/FLU | 19 |
| Reduced* | 17 |
| Treosulfan-based (all TREO/FLU) | 17 |
| Other reduced intensity | 14 |
| With TBI (200–400 cGy) | 4 |
| Other myeloablative | 1 |
| None | 1 |
| Serotherapy used | 38 |
AUC, Area under the curve; BU/CY, busulfan cyclophosphamide; BU/FLU, busulfan fludarabine; MMUD, mismatched unrelated donor; PBSC, peripheral blood stem cell; TBI, total body irradiation; TREO/FLU, treosulfan fludarabine; UCB, unrelated cord blood.
*
Intravenous busulfan dose equivalent to <14 mg/kg oral dosing or busulfan tar-geted to an AUC of <70.000 ng × mL/h.