In older patients with acute myeloid leukemia (AML), the myeloablative busulfan plus fludarabine conditioning regimen is associated with lower transplant-related mortality than busulfan plus cyclophosphamide while retaining potent antileukemic activity, a new study published online ahead of print in the journal The Lancet Oncology has shown.1
Because busulfan plus fludarabine has been proposed to reduce non-relapse mortality in patients with AML undergoing allogeneic hematopoietic stem-cell transplantation (HSCT), researchers sought to compare this combination with busulfan plus cyclophosphamide as a preparative regimen.
For the open-label, multicenter, phase 3 trial, researchers enrolled 252 patients age 40 to 65 with AML in complete remission. Patients were randomly assigned 1:1 to receive intravenous busulfan plus cyclophosphamide or busulfan plus fludarabine.
Results showed that 1-year non-relapse mortality was 17.2% (95% CI: 11.6-25.4) in the busulfan plus cyclophosphamide arm and 7.9% (95% CI: 4.3-14.3) in the busulfan plus fludarabine arm (P=0.026).
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In regard to safety, 23% of patients in the cyclophosphamide group and 21% of patients in the fludarabine group reported grade 3 or higher gastrointestinal adverse events. Infections occurred in 17% and 10% of patients in the cyclophosphamide and fludarabine groups, respectively.
“This regimen should be regarded as standard of care during the planning of allogeneic transplants for such patients,” the authors concluded.
- Rambaldi A, Grassi A, Masciulli A, et al. Busulfan plus cyclophosphamide versus busulfan plus fludarabine as a preparative regimen for allogeneic haemopoietic stem-cell transplantation in patients with acute myeloid leukaemia: an open-label, multicentre, randomised, phase 3 trial [published online ahead of print September 28, 2015]. Lancet Oncol. doi: 10.1016/S1470-2045(15)00200-4.