Post-Myelofibrosis Acute Myeloid Leukemia Effectively Treated with a Combination of Ruxolitinib and 5-Azacytidine

BACKGROUND: Leukemic transformation is an unfavorable event emerging in a minority of patients with myelofibrosis (MF) and carrying a poor prognosis. Patients with post-MF acute myeloid leukemia (AML) may be treated with curative or noncurative intent, but there is no standard of care. The use of hypomethylating agents has been correlated with limited activity and low overall survival rates, while ruxolitinib has been proven to have modest antileukemic activity. CASE PRESENTATION: In this case study, we present the case of a 67-year-old female patient with post-polycythemia vera MF who developed AML and was successfully treated with a combination of 5-azacytidine and ruxolitinib, without any significant toxicity. SUMMARY: To our knowledge, this is the first report of a patient with post-MF AML achieving a complete remission with a combination of ruxolitinib and a hypomethylating agent. This combination is actively studied in phase I/II clinical trials and may be proven effective in this hard-to-treat group of patients.