Clinical perspectives on post-induction maintenance therapy in patients with acute myeloid leukaemia in remission who are ineligible for allogeneic haematopoietic stem cell transplantation

For patients with acute myeloid leukaemia (AML) who achieve complete remission (CR) after induction therapy, subsequent allogeneic haematopoietic stem cell transplantation (allo-HSCT) reduces the risk of relapse. However, not all patients are eligible, warranting effective alternative maintenance strategies. Oral azacitidine is the only non-targeted therapy approved by both the United States (US) Food and Drug Administration and the European Medicines Agency for the maintenance or continued treatment of allo-HSCT-ineligible patients with AML achieving CR or CR with incomplete haematological recovery following induction chemotherapy. Midostaurin and histamine dihydrochloride are approved in Europe as maintenance therapy for AML in remission, and quizartinib is approved in the United States and Europe for the treatment and maintenance of patients with newly diagnosed FLT3-ITD AML. Barriers to maintenance treatment include limited clinical trial data informing appropriate patient and treatment selection, patient preference, financial burden and paucity of real-world data. This article discusses current maintenance treatment guidelines for patients with AML in remission but not proceeding to allo-HSCT and reviews clinical trial data for agents approved for use in remission. Ongoing studies of interest and considerations for future efforts are also discussed.