Study Compares VEN/HMA and Intensive Chemotherapy in Intermediate-Risk AML Patients

What's Happening?

A multicenter real-world study has been conducted to compare the efficacy and safety of venetoclax (VEN) combined with hypomethylating agents (HMAs) versus intensive chemotherapy (IC) in newly diagnosed intermediate-risk acute myeloid leukemia (AML) patients. The study involved 229 patients treated at four hospitals in Hunan Province, China, from January 2016 to February 2025. The research focused on response heterogeneity across genetic subgroups and long-term survival outcomes. Results indicated that VEN/HMA achieved higher overall response and complete remission rates compared to IC, with reduced hematologic toxicity and transfusion requirements. The study also highlighted that patients with specific genetic mutations, such as FLT3-ITD, DNMT3A, or TET2, responded better to VEN/HMA.

Why It's Important?

This study is significant as it provides insights into the potential benefits of VEN/HMA as a frontline induction regimen for intermediate-risk AML patients. The findings suggest that VEN/HMA could offer a safer and more effective treatment option, particularly for patients with certain genetic mutations. This could lead to a shift from age-based to genotype-driven treatment strategies, potentially improving patient outcomes. The reduced toxicity associated with VEN/HMA may also enhance treatment adherence and facilitate the completion of post-remission therapy, which is crucial for long-term disease control.

What's Next?

The study's findings underscore the need for prospective, large-scale clinical trials to validate the efficacy and safety of VEN/HMA in intermediate-risk AML. Future research should focus on mutation-specific response patterns and refine individualized treatment strategies to achieve deeper remission and extend survival outcomes. Additionally, optimizing post-remission interventions, such as consolidation strategies with IC or allo-HSCT, will be essential to maintain long-term remission and address potential VEN resistance.

Beyond the Headlines

The study highlights the importance of tailoring induction therapy based on individual genomic profiles, which could lead to more personalized and effective treatment approaches in AML. The use of minimal residual disease (MRD) as a prognostic marker in AML is also emphasized, suggesting that achieving MRD negativity with VEN/HMA could lead to better overall survival compared to IC. These findings could influence future clinical guidelines and treatment frameworks, promoting a more personalized approach to AML management.