Maintenance Therapy in Acute Myeloid Leukemia: Current Perspectives and Future Directions

The management of acute myeloid leukemia (AML) remains characterized by high relapse rates despite advances in induction and consolidation therapy. Relapse prevention represents a major unmet need, particularly in patients ineligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or at high risk of post-transplant recurrence. This review examines current evidence supporting maintenance strategies following intensive chemotherapy or allo-HSCT, with emphasis on measurable residual disease (MRD)-guided approaches and targeted therapies. We summarize data from randomized and phase II/III trials evaluating hypomethylating agents, FLT3 inhibitors, IDH inhibitors, and immunotherapeutic strategies in post-remission settings. Oral azacitidine (CC-486) demonstrated overall survival benefit in older patients in first complete remission who were not transplant candidates, establishing a standard of care in this population. In FLT3-mutated AML, post-transplant maintenance with sorafenib and gilteritinib reduces relapse risk, with emerging evidence supporting MRD as a predictive biomarker for benefit. Other targeted agents and immunotherapies have shown promising early-phase results, although confirmatory data are limited. Ongoing phase III studies will clarify optimal patient selection, treatment duration, and integration with transplantation, aiming to transform post-remission management from passive surveillance to precision-based relapse prevention.