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

In a recent phase 3 clinical trial presented at the European Hematology Association Congress on June 14, 2025, researchers reported that the addition of dasatinib (Sprycel) to standard induction and consolidation therapy for patients with core-binding factor acute myeloid leukemia (CBF-AML) did not yield significant improvements in event-free survival (EFS) or overall survival (OS). The study involved 204 patients, who were randomly assigned to receive either standard chemotherapy or chemotherapy combined with dasatinib. The results showed 4-year EFS rates of 41% for the standard therapy group and 44% for the dasatinib group, with no statistically significant difference (HR, 0.92; 95% CI, 0.63-1.33; P = .66). Similarly, the OS rates were 76% for standard therapy versus 78% for dasatinib (HR, 0.93; 95% CI, 0.53-1.63; P = .79).

The findings contradict previous single-arm phase 2 studies that suggested dasatinib might enhance treatment effectiveness. Dr. Hartmut Döhner, MD, a leading researcher and professor at the University Hospital Ulm, emphasized the importance of randomized trials in validating treatment efficacy. "The data do not support the encouraging results reported in earlier studies, underscoring the necessity for controlled randomized trials in assessing new agents," Döhner stated during his presentation.

The study's patient demographics revealed a median age of 49.8 years, with 50% of the participants being female. The majority had de novo AML. Genetic assessments for mutations in KIT and FLT3 were also conducted, showing no significant impact on treatment outcomes. The adverse effects (AEs) reported were similar between both treatment arms, with no major differences in hematologic or non-hematologic complications.

These findings reinforce the critical need for rigorous clinical trials in the oncology field, particularly when dealing with promising but unverified therapies. As the research community continues to explore effective treatment modalities for CBF-AML, this study serves as a reminder of the complexities involved in cancer treatment and the necessity for evidence-based approaches to therapy. Looking ahead, the focus will likely shift toward investigating alternative treatment strategies that may better address the unique challenges presented by CBF-AML.