Improvements in Real-World Survival in the Setting of a Recent Paradigm Shift in Acute Myeloid Leukemia Treatment

Therapeutic options for newly diagnosed (ND) acute myeloid leukemia (AML) have increased in recent years, leading to a shift in the treatment paradigm from conventional, intensive chemotherapy toward targeted and less intensive therapy. Since 2017, there has been a surge in FDA approvals for novel therapies, including small molecule inhibitors and new combinations of chemotherapy and targeted drugs. It is unclear what effect these changes have had on overall survival in the real-world clinical setting over time. We retrospectively analyzed 501 patients with ND AML treated at an urban academic cancer center from 2015 to 2022 with respect to survival rates and treatment regimen utilization trends over time, in the context of AML risk category and whether allogeneic stem cell transplantation (HSCT) occurred. Among European LeukemiaNet 2022 intermediate risk ND AML patients who did not undergo HSCT, overall survival and relapse-free survival improved significantly in the early versus later cohort (2015-2018 vs. 2019-2022, respectively). These gains were not observed in the adverse-risk subgroup, where outcomes remain unchanged. First-line use of hypomethylating agent-based therapies increased seven-fold, while reliance on chemotherapy alone decreased by half. These findings highlight a meaningful shift in treatment patterns associated with improved outcomes overall, but an ongoing lack of progress in the highest-risk subgroups.