Multinational phase 3 trial of quizartinib plus chemotherapy for newly diagnosed FLT3-ITD-positive acute myeloid leukemia

Patients with acute myeloid leukemia (AML) positive for internal tandem duplication (ITD) mutations of FLT3 have poor outcomes. Quizartinib is an oral, potent, selective, type 2 FLT3 inhibitor. The multinational phase 3 QuANTUM-First trial evaluation addition of quizartinib versus placebo to first-line chemotherapy for FLT3-ITD-positive AML in adult patients, with or without allo-HCT, followed by continuation monotherapy for up to 3 years. The median overall survival was 31.9 months in the quizartinib group versus 15.1 months in the placebo group (HR 0.78; p=0.032). At least one adverse event was seen in 100% of the patients in the quizartinib group versus 99% of the patients in the placebo group, and at least one grade 3 or higher AE in 92% versus 90%. The authors conclude that quizartinib provides a new, effective, and generally well tolerated treatment option for adult patients with FLT3-ITD-positive newly diagnosed AML.