Decitabine improved response rates and PFS compared with other standard therapies for patients who were ineligible for intensive chemotherapy, with a benefit of almost 4 months in median OS. Although the prognosis in MK AML is usually particularly poor, the median OS in decitabine-treated MK AML patients in the present analysis was comparable to that among MK- patients with unfavorable cytogenetics.
This suggests a potential positive effect of decitabine in patients with MK AML, possibly by reactivating haplo-insufficient tumor suppressor gene mapping on monosomal autosomes.