The euphoria of hypomethylating agents in MDS and AML: is it justified?

While it is logical to use hypomethylating agents to treat patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), trial results with azacitidine and decitabine have been inconsistent. Azacitidine confers a survival benefit in higher-risk MDS patients, while decitabine does not. Neither agent has demonstrated a survival advantage in older AML patients in prospective studies. Reasons for this are explored here along with a recommendation to reconsider clinical trial design endpoints.