Among patients with secondary acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic stem cell transplantation (HSCT), combination granulocyte-colony stimulating factor (G-CSF), decitabine, and busulfan-cyclophosphamide conditioning appears to be an effective strategy, according to research published in The Lancet Haematology.

Although allogeneic HSCT remains the only curative option for MDS, the risk of relapse is notable. Conditioning therapy is used to reduce the risk of relapse in these cases, although the intensity of conditioning regimens varies.

For this study, researchers evaluated the relative effectiveness of combination G-CSF, decitabine, and busulfan-cyclophosphamide with that of busulfan-cyclophosphamide alone as conditioning regimens among patients with MDS-refractory anemia with excess blasts or secondary AML who are undergoing HSCT.

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All patients were enrolled in hospitals in China between 2016 and 2019. Overall, 202 were randomly assigned to receive G-CSF, decitabine, and busulfan-cyclophosphamide (101 patients) or busulfan-cyclophosphamide (101 patients). In the overall cohort, 61% of patients were male sex, and the median ages in the experimental and control groups were 45 and 44 years, respectively.

The median follow-up was 32.4 months. During this period, the 2-year cumulative rate of relapse was 10.9% (95% CI, 5.8-17.9) in the G-CSF group compared with 24.8% (95% CI, 0.19-0.79; P =.011) in the busulfan-cyclophosphamide only group.

In the G-CSF vs busulfan-cyclophosphamide only group, the most common grade 3 or 4 adverse events within 100 days post-HSCT were infection (34% vs 32%, respectively), acute graft-versus-host disease (30% vs 30%), and gastrointestinal toxicity (28% and 29%). No treatment-related deaths were noted, although 11% of patients in the G-CSF group and 13% in the busulfan-cyclophosphamide only group died because of an adverse event.

“In conclusion, our study suggests that G-CSF, decitabine, and busulfan–cyclophosphamide conditioning is a better choice in patients with myelodysplastic leukaemia-[refractory anemia with excess blasts] or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic HSCT compared with busulfan–cyclophosphamide conditioning,” the authors wrote in their report.


Xuan L, Dai M, Jiang E, et al. The effect of granulocyte-colony stimulating factor, decitabine, and busulfan-cyclophosphamide versus busulfan-cyclophosphamide conditioning on relapse in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. Published online January 23, 2023. doi:10.1016/S2352-3026(22)00375-1