The US Food and Drug Administration has approved treosulfan, an alkylating agent, with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients aged one year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Clinical evidence The efficacy of treosulfan was evaluated in a clinical trial involving 570 patients aged 18 to 70 years. The primary efficacy outcome measure was overall survival (OS), the time from randomization to death from any cause.
Compared to busulfan, the hazard ratio for OS was 0.67 in the randomized group, 0.73 in AML patients, and 0.64 in MDS patients. Dosage and administration 10 g/m2 daily on days -4, -3, and -2 in combination with fludarabine 30 mg/m2 daily on days -6, -5, -4, -3, and -2, and allogeneic hematopoietic stem cell infusion on day 0 Adverse…
