Overcoming relapse: prophylactic or pre-emptive use of azacitidine or FLT3 inhibitors after allogeneic transplantation for AML or MDS.

Relapse remains the most critical obstacle in treatment by allogeneic hematopoietic stem cell transplantation (HSCT). Non-relapse mortality has improved annually, but relapse mortality remains high. Post-transplant maintenance treatment, such as hypomethylating agents and FMS-like tyrosine kinase 3 (FLT3) inhibitors, has been investigated for decades as a means of preventing disease relapse after HSCT. Other factors besides the relapse tendency of the primary disease that can affect the transition of estimated disease burden in patients undergoing HSCT are disease status at HSCT (non-remission, remission with minimal/measurable residual disease (MRD), and remission without MRD) and conditioning regimen intensity. Optimal selection of patients at high risk for relapse who can tolerate a long duration of therapy is pivotal for successful post-transplant maintenance therapy. In this review, we provide an overview of current progress in research on post-transplant maintenance treatment using azacitidine or FLT3 inhibitors for preventing disease relapse after HSCT for AML or MDS, and discuss the future outlook in this area.