Combination azacitidine plus venetoclax appears to be safe and active in patients with high-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML), according to phase 1 results published in The Lancet Haematology.

Researchers reported results from the phase 1 portion of a phase 1/2 study ( Identifier: NCT04160052) designed to evaluate azacitidine plus venetoclax in patients with high-risk MDS or CMML.

There were 23 patients enrolled; 17 had MDS, and 6 had CMML. Likewise, 17 patients had not previously received a hypomethylating agent, and 6 had failed prior treatment with a hypomethylating agent. In the overall patient group, the median age was 68 years, and 78% of patients were men.

Continue Reading

The median follow-up was 13.2 months. The overall response rate was 87% (95% CI, 66%-97%).

The maximum tolerated dose was not reached. The recommended phase 2 dose was azacitidine at 75 mg/m2 for 5 days and venetoclax at 400 mg for 14 days.

The most common grade 3 or higher treatment-related adverse events were neutropenia (39%), thrombocytopenia (39%), lung infection (30%), and febrile neutropenia (17%). There were 3 deaths from sepsis, which were deemed unrelated to the study protocol.

“Ultimately, the question of whether the addition of venetoclax to azacitidine improves overall survival in patients with high-risk myelodysplastic syndromes will be answered by the ongoing phase 3, randomised, controlled trial VERONA,” the authors wrote.

Disclosures: The study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 


Bazinet A, Darbaniyan F, Jabbour E, et al. Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: Phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1-2 study. Lancet Haematol. Published online September 2, 2022. doi:10.1016/S2352-3026(22)00216-2

This article originally appeared on Hematology Advisor