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 mutation
- 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.


