Abstract
BACKGROUND: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the sole curative option for myeloid malignancies, though high toxicity from conditioning regimens and complications like graft-versus-host disease (GVHD) limit its success. The potential benefit of incorporating decitabine (DAC) into conditioning regimens for acute myeloid leukemia (AML) and intermediate-to-high-risk myelodysplastic syndromes (MDS) patients in remission remains unclear. METHODS: We conducted a retrospective, single-center study analyzing data from January 2016 to December 2020 at the First Affiliated Hospital of Ningbo University with a median follow-up of 45.05 months (range, 1-96 months). Outcomes were compared between patients receiving DAC+HSCT versus HSCT alone, with primary endpoints of 5-year overall survival (OS), progression-free survival (PFS), and relapse rate. Secondary analyses examined outcomes by remission status (CR1 vs. others) and age subgroups (< 31.5 years). Immune cell subsets (CD3-CD56+ NK cells) were evaluated for GVHD correlation. RESULTS: The DAC+HSCT group exhibited 5-year OS of 51.9% and PFS of 46.1%, compared to 67% OS and 56.5% PFS in the HSCT-only group. The 5-year relapse rate was 16.9% for DAC+HSCT versus 23.2% for HSCT alone. DAC did not significantly improve outcomes in complete remission (CR1) patients but improved OS and PFS in patients under 31.5 years of age. Elevated CD3-CD56+ NK cells in the DAC+HSCT group were associated with higher incidence of severe acute GVHD (aGVHD). CONCLUSION: While DAC conditioning did not provide overall survival benefit for AML/MDS patients undergoing allo-HSCT, it improved outcomes in younger individuals (< 31.5 years). Higher NK cell proportions may serve as a potential biomarker for early aGVHD intervention, warranting further investigation into risk-stratified conditioning approaches.