Abstract

Summary This prospective clinical study aimed to evaluate the efficacy and safety of the pre‐emptive treatment modality of azacitidine in combination with interferon‐α (IFN‐α) in AML/MDS patients post‐transplantation. Forty‐seven patients aged 17–62 were enrolled with 14 patients having completed the planned 12 cycles. Following initiation, 72.3% responded positively after the first cycle, peaking at 77.2% by the fifth cycle. Notably, 24 patients maintained sustained responses throughout a median follow‐up of 1050 days (range, 866–1234). Overall survival, leukaemia‐free survival and event‐free survival probabilities at 3 years were 69.5%, 60.4% and 35.7% respectively. Cumulative incidences of relapse and non‐relapse mortality were 36.5% and 4.3% respectively. Multivariate analysis identified that receiving pre‐emptive treatment for fewer than six cycles and the absence of chronic graft‐versus‐host disease after intervention was significantly associated with poorer clinical outcomes. The combination of azacitidine with IFN‐α was well‐tolerated with no observed severe myelotoxicity, and the majority of adverse events were reversible and manageable. In conclusion, the use of azacitidine in conjunction with IFN‐α as pre‐emptive therapy is a safe and effective treatment to prevent disease progression in AML/MDS patients with MRD positivity post‐allo‐HSCT.