A recent retrospective study presented at the 2024 ASH Annual Meeting has shed light on the potential benefits of combining venetoclax (Venclexta) with hypomethylating agents (HMAs) for treating myelodysplastic syndrome (MDS). The study found that this combination therapy significantly outperformed HMAs alone in adult patients with MDS.
Venetoclax and HMAs: A Powerful Combination for MDS
The retrospective study, which included data from 13 academic medical centers and 454 patients, revealed that the combination of venetoclax and HMAs achieved a complete response (CR) rate of 33%, compared to 12% with HMAs alone. Furthermore, the marrow CR rate was 40% for the combination therapy versus 27% for HMAs alone (P < 0.001). These findings suggest that venetoclax may significantly improve treatment outcomes for MDS patients.
Beyond initial treatment, the combination therapy demonstrated encouraging results even after HMA failure. Patients who received venetoclax after HMA failure achieved a CR and marrow CR rate of 10% and 32%, respectively.
Improved Survival and Reduced Adverse Events
The study also highlighted the positive impact of venetoclax on event-free survival (EFS). Patients receiving the combination therapy experienced a statistically significant improvement in EFS compared to those receiving HMAs alone (HR, 0.59; 95% CI, 0.44-0.78; P < 0.001).
While the combination therapy did carry some risks, the incidence of tumor lysis syndrome (TLS) was low, with only 1 patient experiencing clinical TLS. Notably, neutropenic fever was the most common adverse event observed, occurring in 34.2% of patients receiving upfront venetoclax, 22.5% in the HMA monotherapy arm, and 44.5% in the post-HMA venetoclax arm.
Looking Ahead: Continued Research and Hope for MDS Patients
These findings from the retrospective study provide promising insights into the potential of venetoclax combined with HMAs for treating MDS. Further research, including larger prospective trials, is needed to confirm these findings and explore optimal treatment strategies. However, the preliminary results offer hope for improved outcomes and quality of life for patients living with MDS.
References
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Murthy GSG, Ball S, Feld J, et al. Clinical utilization and outcomes of hypomethylating agents and venetoclax in patients with myelodysplastic syndrome- a multicenter retrospective analysis. Blood. 2024;144(suppl 1):3206.doi:10.1182/blood-2024-211623
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Zeidan AM, Borate U, Pollyea DA, et al. A phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes. Am J Hematol. 2023;98(2):272-281. doi:10.1002/ajh.26771