Table 1.
Description of patient population.a
| Total number of patients | 729 |
| Age, y | 7.1 ± 6.0 (0.1–20.0) |
| Dosing weight, kg | 28.7 ± 23.0 (2.65–117.8) |
| Sex | |
| Male | 402 |
| Female | 325 |
| Not reported | 2 |
| Diagnosis | |
| AML/MDS | 264 |
| CID | 88 |
| CML/JMML | 65 |
| Pediatric solid tumor | 43 |
| Histiocytic disorder | 39 |
| Acute lymphoblastic leukemia | 37 |
| Metabolic storage disease | 36 |
| Bone marrow failure | 30 |
| Sickle cell anemia | 28 |
| Thalassemia | 22 |
| AML/MDS, secondary | 16 |
| Aplastic anemia | 15 |
| Granulocyte disorder | 15 |
| Osteopetrosis | 12 |
| Lymphoma | 7 |
| Hodgkin lymphoma | 6 |
| Myeloproliferative disorder | 5 |
| Paroxysmal nocturnal hemoglobinuria |
1 |
| Initial dose for children ≤12 kg,b mg/kg | |
| 0.8 | 48 |
| 1.0 | 97 |
| 1.1c | 37 |
| Other | 25 |
| Initial dose for children >12 kg,b mg/kg | |
| 0.8c | 333 |
| 0.9 | 23 |
| 1.0 | 94 |
| Other | 28 |
Abbreviations: AML/MDS, acute myelogenous leukemia/myelodysplastic syndrome; CID, combined immunodeficiency disorders; CML/JMML, chronic myelogenous leukemia/juvenile myelomonocytic leukemia.
a
Data presented as n or as mean ± standard deviation (range).
b
For children receiving Q6hr busulfan; dose rounded to the nearest 0.1 mg/kg; more detailed description within Supplemental Table S2.
c
Dose per Busulfex package insert.