Compared with placebo and azacitidine, the combination of ivosidenib and azacitidine prolongs event-free survival for patients with newly diagnosed IDH1-mutated acute myeloid leukemia who are ineligible for induction chemotherapy, according to a study published in the April 21 issue of the New England Journal of Medicine.

Pau Montesinos, MD, PhD, from Hospital Universitari i Politècnic La Fe in Valencia, Spain, and colleagues conducted a phase 3 trial involving patients with newly diagnosed IDH1-mutated acute myeloid leukemia who were ineligible for intensive induction chemotherapy. Patients were randomly assigned to receive oral ivosidenib and subcutaneous or intravenous azacitidine or matched placebo and azacitidine (72 and 74 patients, respectively).

The researchers found that event-free survival was significantly longer in the ivosidenib-and-azacitidine group than in the placebo-and-azacitidine group at a median follow-up of 12.4 months (hazard ratio for treatment failure, relapse from remission, or death, 0.33). The estimated probability that a patient would remain event-free at 12 months was 37 and 12 percent in the ivosidenib-and-azacitidine group and the placebo-and-azacitidine group, respectively. Median overall survival was 24.0 and 7.9 months in the ivosidenib-and-azacitidine group and placebo-and-azacitidine group, respectively (hazard ratio for death, 0.44).

"Because this trial showed a robust improvement in all efficacy end points, it becomes important to consider the positioning of this new option in the current treatment landscape, which includes venetoclax-based regimens," the authors write.

The study was funded by Agios Pharmaceuticals. Servier Pharmaceuticals has completed the acquisition of the Agios oncology business.

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