The authors of this phase Ib trial evaluated the oral IDH1 inhibitor ivosidenib in combination with azacitidine in 23 patients with newly diagnosed acute myeloid leukemia ineligible for intensive induction chemotherapy. The overall and complete response rates were 78% and 61%, respectively. The median duration of response and median overall survival were not yet reached with 16 months’ median follow-up. The 12-month overall survival estimate was 82%. Treatment was well-tolerated with no dose-limiting toxicities.
Ivosidenib plus azacitidine was well tolerated, with an expected safety profile consistent with monotherapy with each agent. Responses were deep and durable, with most complete responders achieving mIDH1 mutation clearance.