Phase 3 trial of adding G-CSF and decitabine to busulfan-cyclophosphamide conditioning for alloHSCT in MDS/AML

Relapse remains high after allogeneic hematopoietic stem cell transplantation (alloHSCT) for myelodysplastic syndrome-refractory anemia with excess blasts (MDS-RAEB) or secondary acute myeloid leukemia evolving from MDS (sAML). A multicenter phase 3 study in China evaluated addition of G-CSF and decitabine to the busulfan-cyclophosphamide alloHSCT conditioning regimen for these patients. Two-year cumulative relapse rate (primary endpoint) was 10.9% with G-CSF and decitabine added to busulfan-cyclophosphamide conditioning versus 24.8% with busulfan-cyclophosphamide only conditioning (HR 0.39; p=0.011). There were no significant differences in adverse events between the two groups. The authors conclude that G-CSF, decitabine, and busulfan-cyclophosphamide conditioning is a better choice than busulfan-cyclophosphamide conditioning for patients undergoing alloHSCT for MDS-RAEB or sAML.