Trial in Progress: A Phase 3, Randomized, Double-Blind Study of Midostaurin in Combination with Chemotherapy and as Single-Agent Maintenance Therapy in Newly Diagnosed Patients with FLT3 Mutation–Negative Acute Myeloid Leukemia (AML)

Midostaurin, a multikinase inhibitor, targets wild-type (WT) and mutated FLT3 and other kinases involved in AML pathogenesis. In RATIFY, midostaurin plus chemotherapy and as single-agent maintenance therapy improved overall survival (OS) and event-free survival (EFS) in adults with FLT3 mutation–positive AML; benefit was seen regardless of FLT3-mutant-to-WT signal ratio and mutation type. FLT3 is overexpressed in ≥70% of patients with AML (independent of FLT3-mutations). Preclinical and clinical evidence suggests a role for midostaurin plus chemotherapy in FLT3 mutation–negative (FLT3-MN) AML.