Phase I/II trial shows high response rates in high-risk myelodysplastic syndromes, indicating potential benefits of azacitidine-venetoclax combinations.
Key Points
To evaluate the safety and efficacy of azacitidine and venetoclax in untreated high-risk myelodysplastic syndromes and chronic myelomonocytic leukemia.
Conducted a phase 1/2 trial at The University of Texas MD Anderson Cancer Center
Patients with IPSS intermediate-2 or high-risk myelodysplastic syndromes were enrolled
Used a 3+3 design to determine venetoclax dosing alongside azacitidine administration
Overall response rate was 93%, with 30% achieving complete remission according to IWG 2006 criteria
Median overall survival was 16.1 months with notable variance based on treatment and molecular profile
Combination therapy resulted in significant rates of adverse events but managed to demonstrate encouraging efficacy