Dr Jain on the VERONA Study in Higher-Risk MDS

Akriti Jain, MD, Department of Hematology and Medical Oncology, Cleveland Clinic, discusses the phase 1b VERONA study (NCT02942290) of venetoclax (Venclexta) with azacitidine (Vidaza) to assess change in complete remission (CR) and overall survival (OS) in patients with treatment-naive higher-risk myelodysplastic syndromes (MDS).

The high-risk MDS patient population is currently the focus of much anticipation surrounding the VERONA trial, which is evaluating the combination of venetoclax and azacitidine, Jain begins. Updated results from the phase 1b study were presented at the 2023 ASH Annual Meeting. In this investigation, 107 patients with high-risk MDS were enrolled, and the outcomes demonstrated a notable modified overall response rate of 80.4% (95% CI, 71.6%-87.4%), she reports. Among patients who responded to treatment, 50.5% (95% CI, 40.6%-60.3%) achieved marrow complete remission (CR), and within this subgroup, 37.0% (95% CI, 24.3%-51.3%) also experienced hematological improvement—a promising finding indicating potential disease control and symptom relief, Jain says. Jain added that 29.9% (95% CI, 21.4%-39.5%) of patients achieved CR, reflecting a significant treatment response.

The median OS observed in this study was 26.0 months (95% CI, 18.1-51.5), with a CR duration reaching 16.6 months (95% CI, 10.0-not reached). These results underscore the potential of combining venetoclax with azacitidine in the high-risk MDS setting to achieve meaningful clinical responses and extend survival outcomes, she explains.

This area of research holds significant promise for patients with high-risk MDS, especially in facilitating successful transplantation or improving long-term outcomes by prolonging OS and response duration, Jain continues. The data from the VERONA trial are eagerly awaited within the medical community as they hold the potential to further enhance the understanding of treatment approaches for patients with MDS, she notes.

The integration of combination treatment approaches represents a promising strategy in the pursuit of more effective and personalized therapies for high-risk MDS, according to Jain. The results from ongoing clinical trials, including the VERONA study, will be instrumental in guiding clinical practice for patients with MDS, Jain concludes.