Real-world data with ivosidenib-azacitidine as 1st line treatment for unfit AML patients

At ASH 2025, Prof Dr Joshua Zeidner, Associate Professor of Medicine at the University of North Carolina Lineberger Comprehensive Cancer Center, presented a poster reporting results from a real-world analysis of frontline treatment with ivosidenib plus azacitidine (IVO-AZA) in older, unfit patients with IDH1-mutated acute myeloid leukemia (AML). This analysis addresses an important clinical question, as the optimal initial treatment strategy for this patient population remains uncertain.

The study used data from the U.S.-based TriNetX electronic health record database. Adult patients with newly diagnosed AML treated over an approximately five-year period were identified, and those who received the combination of IVO-AZA in routine clinical practice were analyzed. A total of 23 patients met the inclusion criteria.

Despite the limited sample size, outcomes were broadly consistent with those reported in prospective clinical trials, including the randomized phase III AGILE study. Median overall survival was not reached at the time of analysis. The estimated 1-year overall survival rate approached 80%, suggesting substantial clinical benefit in this older, unfit population. While detailed response assessments and molecular subgroup analyses were not available within this dataset, the observed survival outcomes provide supportive evidence for the effectiveness of IVO-AZA outside the context of controlled clinical trials.