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ESH AL 2026 | The evolving treatment landscape for patients with IDH1-mutated AML
Eytan Stein, MD, Memorial Sloan Kettering Cancer Center, New York, NY, comments on the treatment of patients with IDH1-mutated acute myeloid leukemia (AML), highlighting the use of the IDH1 inhibitor ivosidenib in combination with azacitidine for older patients unfit for intensive chemotherapy. He also discusses the potential benefits of combining ivosidenib with azacitidine and venetoclax, summarizing recent data with this combination. This interview took place at the 5th How to Diagnose and Treat: Acute Leukemias meeting of the European School of Hematology (ESH AL) in Mandelieu-La Napoule, France.
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Transcript
We had a really great session today. We talked about how we treat patients with IDH1-mutated acute myeloid leukemia. And we can use the IDH1 inhibitor, ivosidenib, for patients with newly diagnosed acute myeloid leukemia who are older and unfit for intensive chemotherapy, where in a randomized phase 3 placebo-controlled trial, those patients had median overall survival of 29.3 months when you combine ivosidenib with azacitidine compared to only around eight months for those patients who got azacitidine alone...
We had a really great session today. We talked about how we treat patients with IDH1-mutated acute myeloid leukemia. And we can use the IDH1 inhibitor, ivosidenib, for patients with newly diagnosed acute myeloid leukemia who are older and unfit for intensive chemotherapy, where in a randomized phase 3 placebo-controlled trial, those patients had median overall survival of 29.3 months when you combine ivosidenib with azacitidine compared to only around eight months for those patients who got azacitidine alone. So really a dramatic difference in the median overall survival. We also talked a little bit about what might happen if you combine three drugs together. You give ivosidenib, azacitidine with venetoclax as a triplet. And in the published data that was recently published in the Journal of Clinical Oncology, it looks like those patients have really, really spectacular outcomes with overall survival that perhaps are going to end up being better than with azacitidine and venetoclax or with azacitidine alone with venetoclax. So to investigate this, there’s now a randomized placebo-controlled phase three trial, which is called Evolve One, where patients are being randomized with newly diagnosed IDH1 mutant AML, either to receive azacitidine, ivosidenib, and placebo, or azacitidine, ivosidenib, and venetoclax. That’s really exciting. On the intensive chemotherapy front, we have some data recently showing that adding ivosidenib to intensive chemotherapy is a safe thing to do and leads to good outcomes. Three-year overall survival is 67%. We’re awaiting the results of another randomized phase 3 trial combining ivosidenib with intensive chemotherapy compared to intensive chemotherapy with placebo, and we’re hoping to have that data within the next year. So a lot of excitement around ivosidenib, around IDH1 inhibitors generally, and a lot of new data that’s going to come out hopefully in the next few years.
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