Dasatinib Fails to Enhance Survival in Core-Binding Factor AML Patients

In a significant development presented at the 2025 European Hematology Association Congress, dasatinib, a tyrosine kinase inhibitor, did not demonstrate improved efficacy in patients with core-binding factor acute myeloid leukemia (CBF-AML) compared to standard therapies. The findings stem from a phase 3 trial (NCT02013648) involving 204 patients, which sought to assess the drug’s impact on event-free survival (EFS) and overall survival (OS).

The trial revealed that patients receiving standard therapy, which included daunorubicin and cytarabine, had a 4-year EFS rate of 41%, slightly outperforming the dasatinib cohort, which reported a 44% EFS rate, with a hazard ratio (HR) of 0.92 (95% CI, 0.63-1.33; P = .66). Similarly, the OS rates were comparable, with standard therapy achieving 76% versus 78% in the dasatinib group (HR, 0.93; 95% CI, 0.53-1.63; P = .79).

Dr. Hartmut Döhner, MD, a notable figure in hematology and director at the University Hospital Ulm, stated, 'In patients with CBF-AML, the addition of dasatinib to induction and consolidation chemotherapy followed by a 12-month maintenance treatment did not improve EFS and OS.' These results starkly contrast prior phase 2 studies that indicated more favorable outcomes, underscoring the necessity for controlled randomized trials to evaluate new treatments effectively.

The research, which spanned from August 2014 to February 2021, randomized patients between two arms: one receiving standard chemotherapy and the other receiving dasatinib in addition to the standard regimen. The median age of participants in both arms was around 49 years, with a majority being female. Notably, the study identified that adverse effects were prevalent, with significant rates of thrombocytopenia, leukopenia, and pneumonia recorded, highlighting the importance of continuous monitoring and evaluation of treatment regimens.

The trial also assessed the rates of complete remission (CR) and complete remission with incomplete hematologic recovery (CRi). The standard therapy arm achieved a CR rate of 64.7%, whereas dasatinib's arm recorded a lower CR rate of 55%. These figures suggest that dasatinib's addition may not provide the anticipated therapeutic benefits.

This trial's findings contribute to the ongoing discourse on effective treatments for CBF-AML, a subtype of AML characterized by specific genetic abnormalities. The results may prompt a reevaluation of treatment protocols and highlight the significance of evidence-based approaches in oncology.

As the medical community deliberates these results, the implications for clinical practice and future research are profound, particularly regarding the development of novel therapies for leukemia patients. The need for further studies remains paramount to ensure that patients receive the most effective treatments available.

In conclusion, while dasatinib has shown promise in other contexts, its role in CBF-AML treatment appears limited based on this comprehensive analysis. Future investigations will be essential to identify potential strategies that may enhance patient outcomes in this challenging area of hematologic oncology.