Ivosidenib Plus Azacitidine Significantly Improves Survival in IDH1-Mutated AML: Long-Term Results from the AGILE Study

Highlights

• Ivosidenib plus azacitidine extended median overall survival (OS) to 29.3 months versus 7.9 months with placebo plus azacitidine in newly diagnosed IDH1-mutated AML patients unfit for intensive chemotherapy.
• The combination regimen led to a transfusion independence rate of 53.8% compared to 17.1% with placebo.
• Lower rates of febrile neutropenia and infections were observed, though neutropenia and bleeding events increased with ivosidenib-azacitidine.
• These results support the combination as a new standard of care in this population, offering the longest OS reported to date.

Clinical Background and Disease Burden