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Results from the phase 3 VERONA trial were presented at the 2025 Society of Hematologic Oncology Annual Meeting.
Results from the phase 3 VERONA trial were presented at the 2025 Society of Hematologic Oncology Annual Meeting.
Patients with revised international prognostic scoring system intermediate and high-risk myelodysplastic syndrome (MDS) did not experience an overall survival (OS) improvement from the treatment combination of Venclexta (venetoclax) plus azacitidine when compared with placebo, study results have shown.
Results from the phase 3 VERONA trial were presented at the 2025 Society of Hematologic Oncology Annual Meeting.
In the combination arm, the median OS was 22.18 months versus 21.68 months in the placebo arm. The modified overall response rate (ORR) in the Venclexta/azacitidine arm was 76.2%. The marrow complete response (CR) rate was 57.8%, the partial response (PR) rate was 0.4% and the CR rate was 18%. In the placebo arm, the modified ORR was 57.7%, the marrow CR rate was 37.5% and the CR rate was 20.2%.
Glossary:
Overall survival: the time a patient lives, regardless of disease status.
Overall response rate: patients who responded partially or completely to treatment.
Neutropenia: low count of neutrophils, a type of white blood cell.
Anemia: low count of healthy red blood cells.
Thrombocytopenia: low count of platelets.
The overall hematological improvement in the combination arm was 49.4% compared with 41.2% in the placebo arm. The transfusion independence rates were 55.7% versus 33.6%, respectively. The median duration of transfusion independence was 183 days vs 294 days.
“Subgroup analyses [showed] [Venclexta] plus azacitidine trended toward an mOR benefit versus placebo plus azacitidine for patients with more than 5% to less than 20% bone marrow blasts for ASXL1, TP53 and RUNX1 mutations at baseline,” Dr. Guillermo Garcia-Manero, chair ad interim in the Department of Leukemia, Division of Cancer Medicine, fellowship program director in the Department of Leukemia, and Dr. Kenneth B. McCredie Chair in Clinical Leukemia Research among other titles, at The University of Texas MD Anderson Cancer Center in Houston, said during the presentation.
A total of 509 patients were randomly evenly assigned to either the combination or placebo arms. Patients were given Venclexta at 400 mg by mouth daily on days 1 to 14 plus azacitidine at 75 mg/m2 intravenously or subcutaneously for seven of the first nine days. The placebo plus azacitidine arm matched the combination arm’s dosing. Patients continued treatment until relapse, progressive disease, hematopoietic stem cell transplant (HSCT) or unacceptable toxicity. Patients then transitioned to follow-up.
The median follow-up was 41.2 months, the median duration of treatment was 6.4 months and the median number of cycles was six.
No new safety signals were observed. The most common any-grade treatment-emergent side effects included neutropenia (77.3% versus 60.2%), thrombocytopenia (66.3% versus 58.9%), anemia (44.7% versus 38.2%) and constipation (42.4% versus 50.4%) between the combination and placebo arms of the trial, respectively.
Serious side effects leading to treatment discontinuation occurred in 41.6% of patients in the combination arm and 55.3% of patients in the placebo arm, while dose interruption was noted in 78.8% versus 67.9% of patients and dose reduction of Venclexta or placebo was found in in 12.5% versus 6.9% while azacitidine was 48.6% versus 27.2%.
Side effects leading to death included disease progression (1.6% versus 0.8%), septic shock (1.2% versus 0.8%), sepsis (0.4% versus 0.8%) and acute myeloid leukemia transformation (0.4% versus 0.8%) among patients in the combination and placebo arms, respectively. The most common cause of death was disease progression (39.2% versus 40.7%) and side effects (10.6% versus 11.4%).
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