Recent advances in the treatment of FLT3-mutated acute myeloid leukemia

FMS-like tyrosine kinase 3 (FLT3) mutations are among the most common driver alterations in acute myeloid leukemia (AML), occurring in approximately 25-30% of patients, and have historically been associated with high relapse rates and poor survival. The therapeutic landscape for FLT3-mutated AML has evolved substantially with the development of FLT3-targeted agents. In newly diagnosed AML, the addition of midostaurin or quizartinib to intensive induction chemotherapy has demonstrated significant improvements in overall survival (OS), event-free survival, and relapse-free survival, establishing these regimens as current standards of care. In the relapsed or refractory setting, gilteritinib has become the preferred treatment based on superior survival and remission outcomes compared with salvage chemotherapy. Molecular studies have identified genomic factors associated with response and resistance to FLT3 inhibition, highlighting the importance of co-occurring mutations in treatment selection and prognosis. Maintenance therapy following allogeneic hematopoietic stem cell transplantation has emerged as a promising strategy, with evidence supporting the use of sorafenib and gilteritinib to reduce relapse risk and improve posttransplant outcomes. Recent clinical investigations have focused on novel combination approaches, particularly triplet regimens, incorporating FLT3 inhibitors, venetoclax, and hypomethylating agents, which have demonstrated encouraging responses and survival outcomes. Next-generation FLT3 inhibitors, including crenolanib and dapolsertib, are being evaluated in ongoing studies. These advances continue to improve outcomes in patients with FLT3-mutated AML and are expected to further refine treatment strategies across disease settings.