Ivosidenib (mutant IDH1 inhibitor) + azacitidine drives a 74-month complete remission in IDH1-mutated elderly AML (case report)

Ivosidenib is an oral small-molecule inhibitor of mutant IDH1 used with the hypomethylating agent azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy (IC). In a single-patient case report from the Phase 3 AGILE study (NCT03173248), an 81-year-old woman diagnosed in 2019 with IDH1-mutated AML achieved complete response after 3 cycles (blasts 1%) and remained on therapy at 74 months (age 88) with dose-reduced ivosidenib/azacitidine and good functional status; molecularly, IDH1 and RUNX1 cleared on treatment, while ASXL1 later emerged. Safety details were qualitative only (“well tolerated”), so this is hard to benchmark against broader AGILE safety (and in practice, against aza/ven’s cytopenias/infections), but the duration of response is far beyond typical medians reported for unfit AML regimens (e.g., AGILE reported median OS 24.0 months with ivosidenib+azacitidine vs 7.9 months with azacitidine alone; VIALE-A reported median OS 14.7 months with azacitidine+venetoclax).