Key clinical point : A myeloablative, fractionated busulfan (f-Bu) regimen reduces relapse and boosts survival without increasing nonrelapse mortality (NRM) in older patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
Major finding : At 2 years, the f-Bu group had a significantly better progression-free survival than the nonfractionated, lower dose busalfan (Bu) group (45% vs 24%; hazard ratio [HR], 0.6; P = .004), attributed to a significant reduction in progression (34% vs 59%; HR, 0.5; P = .003) without an increase in NRM (21% vs 15%; HR, 1.4; P = .3). Overall survival also improved in the f-Bu group vs the Bu group (51% vs 31%, HR, 0.6; P = .01).
Study details : In an open-label, phase 2 trial, patients with AML or MDS were randomly assigned to either myeloablative f-Bu over 2 weeks (n = 84; median age, 65 years) or a standard Bu regimen over 4 days (n = 78; median age, 66 years).
Disclosures: This study was supported in part by a grant from the National Cancer Institute. Q Bashir, C Hosing, K Rezvani and UR Popat reported relationships with various pharmaceutical companies and/or research organizations. The remaining authors declared no conflicts of interest.
Source: Oran B et al. Cancer. 2021 Jan 20. doi: 10.1002/cncr.33383