Phase 1 results of azacitidine plus venetoclax for high-risk myelodysplastic syndromes or chronic myelomonocytic leukemia

Therapies beyond hypomethylating agents such as azacitidine are needed in high-risk myelodysplastic syndromes (MDS). Venetoclax is an orally bioavailable small molecule BCL-2 inhibitor that is synergistic with hypomethylating agents. A phase 1-2 study at MD Anderson Cancer Center (Houston, TX) evaluates azacitidine plus venetoclax for treatment-naïve and relapsed or refractory high-risk MDS or chronic myelomonocytic leukemia (CMML). Phase 1 results of the study showed that azacitidine plus venetoclax is safe and shows encouraging activity (ORR 87%; 95% CI 66-97) in patients with high-risk MDS or CMML.