A Gentler Approach for Treatment of Younger Patients With AML? | Amir Fathi, MD | EHA 2025 #AML

Hi, my name is Dr. Amir Fatih. I'm a leukemia specialist practicing at Massachusetts General Hospital in Boston, Massachusetts. I direct the leukemia program there. I'm also associate professor of medicine at Harvard Medical School. I run clinical trials on acute leukemias and bone marrow cancers. So I would like to talk to you today about a recent clinical trial that we have concluded in terms of accrual. It's a phase two randomized study of looking at azacytidine and venetoclax, a more gentle combination regimen already approved in older patients with AML. But in this study, we are actually comparing it to the standard of care for younger patients with AML, which is intensive chemotherapy. Intensive chemotherapy comes at a huge cost, financial, physical, psychiatric. Generally speaking, it's quite toxic, but it has been used for several decades and it's the current standard of care for younger patients with AML. We wanted to see whether azacytidine and venetoclax, a more gentle approach, which has shown significant promise in older patients, can show similar, if not better, promise in younger patients. And therefore, we're conducting a randomized phase two study of traditional intensive induction chemotherapy versus azacytidine and venetoclax in younger intensive therapy eligible patients. The primary endpoint of the study is event-free survival, but other very key endpoints are also included, overall survival, MRD, transplant bridging, cost of health care, quality of life, a lot of important factors in terms of how we can potentially improve the treatment of AML patients, make it more tolerable, and so on. The trial is currently finished accrual and we hope to have data that we can present in the very near future.