Table 1.
Patients’ characteristics. Continuous data are presented as means with standard deviations and minimum-maximum ranges in brackets. Categorical data are presented as counts
| Characteristic | Value |
|---|---|
| Age [years] | 7.8 ± 4.9 (0.4–15) |
| Bodyweight [kg] | 26.9 ± 15.7 (7.7–52) |
| Body surface area [m2] | 0.95 ± 0.44 (0.25–1.63) |
| Boys/girls [n] | 12 / 3 |
| Total daily treosulfan dose and infusion length (n) | |
| 10 g/m2–1 h | 1 |
| 12 g/m2–1 h | 4 |
| 12 g/m2–2 h | 4 |
| 14 g/m2–2 h | 6 |
| Creatinine clearance [ml/min] (n = 8) | 123 ± 60 (71–239) |
| Diagnosis | |
| Hematological malignancies | |
| ALL | 4 |
| AML | 1 |
| CML | 1 |
| Solid tumors | |
| NBL | 2 |
| ES | 2 |
| Non-malignant disorders | |
| X-ALD | 2 |
| DBA | 1 |
| SCN | 1 |
| WAS | 1 |
X-ALD adrenoleukodystrophy, ALL acute lymphoblastic leukemia, AML acute myeloid leukemia, CML chronic myeloid leukemia, DBA Diamond-Blackfan anemia, ES Ewing’s sarcoma, NBL neuroblastoma, SCN severe congenital neutropenia, WAS Wiskott-Aldrich syndrome