Low-Intensity Pretransplant Therapy in Acute Myeloid Leukemia: A 13-Year Single-Center Experience

Allogeneic hematopoietic cell transplantation (allo-HCT) following complete remission (CR) after induction chemotherapy has traditionally been the only curative approach for high-risk acute myeloid leukemia (AML). Recent advances have expanded allo-HCT eligibility to older patients. However, the requirement of achieving CR before transplant is increasingly being challenged. We retrospectively analyzed outcomes of 106 AML patients who underwent allo-HCT between 2010 and 2023 at our center, regardless of remission status. Patients received either hypomethylating agents (HMA, n = 42), HMA plus venetoclax (HMA + Ven, n = 24), or no prior therapy (upfront, n = 40). CR at the time of transplant was achieved in 19% of HMA and 38% of HMA + Ven patients. One-year non-relapse mortality (NRM) was significantly higher in the HMA group (26.2%) compared to HMA + Ven (4.2%, p = 0.0062) and upfront (5.0%, p = 0.0061) groups. However, the one-year cumulative incidence of relapse was similar across cohorts (HMA 19.1%, HMA + Ven 16.7%, upfront 15.0%). Three-year overall survival was 47.6% (HMA), 61.7% (HMA + Ven), and 71.8% (upfront). These findings support the feasibility of allo-HCT in AML patients treated with low-intensity pretransplant regimens, even without prior CR, and highlight the need for prospective trials to to avoid excluding patients with active disease from potentially curative options.