Triplet combinations improve outcomes for elderly patients with IDH-mutant AML
MD Anderson Research Highlight June 24, 2025
New targeted therapies have improved treatment options for elderly patients with IDH-mutant acute myeloid leukemia (AML) who are not eligible for intensive chemotherapy, with two double therapy regimens approved as frontline therapies. However, most patients either do not benefit or will experience a relapse. Therefore, researchers led by Courtney DiNardo, M.D., evaluated new triplet regimens in 60 newly diagnosed, elderly patients with IDH-mutated AML who were ineligible for chemotherapy, including:
- Azacitidine, venetoclax and ivosidenib for patients with IDH1 mutations
- Oral decitabine, venetoclax and ivosidenib for patients with IDH1 mutations
- Oral decitabine, venetoclax and enasidenib for patients with IDH2 mutations
The regimens were well tolerated with similar safety profiles to previous doublet regimens. The therapies achieved complete remissions in 92% of patients and an overall response rate of 95%. After more than two years of follow-up, nearly 70% of patients were still alive, and relapse rates were lower than anticipated, especially in patients who had not received prior therapy. Researchers continue to examine patient outcomes, and this study has informed a large Phase III randomized trial that is currently enrolling patients. Learn more in the Journal of Clinical Oncology.
AML outcomes have improved substantially over the past decade, but most older patients still are not cured. Compared to either doublet regimen alone, our experience with triplet regimens including a hypomethylating agent, venetoclax and an IDH inhibitor demonstrates dramatic and durable responses, with no evidence of increased toxicity. We are hopeful this regimen may prove to be an optimal strategy for newly diagnosed patients with IDH-mutated AML in the near future