Venetoclax and hypomethylating agents (HMAs) induce high response rates in MDS, including patients after HMA therapy failure.

Treatment-naive and relapsed/refractory MDS patients receiving venetoclax and HMAs have an ORR of 59% with 63% of responders proceeding to transplant. Allogeneic stem cell transplantation after treatment with venetoclax in combination with HMA is associated with prolonged survival.