• MPNRF | April 28, 2021

    By Raajit Rampal M.D. Ph.D., Assistant Attending Physician, Leukemia Service
    Memorial Sloan-Kettering Cancer Center

    The treatment of patients who have an MPN which evolves into an accelerated or blast-phase (greater than 10% or 20% blast-cells in the blood or bone marrow, respectively) remains a challenge. Patients with blast-phase or accelerated-phase disease are often treated with chemotherapy that is typically given to patients with a disease called acute myeloid leukemia (AML).  However, most patients don’t respond to this therapy. Previous studies have demonstrated that ruxolitinib, as well as a class of drugs called hypomethylating agents, may help patients with blast-phase or accelerated disease. In work performed in the laboratory, we recently showed that these drugs might work together to improve efficacy, and therefore conducted a phase I trial testing the combination of Ruxolitinib and Decitabine (a hypomethylating agent) in MPN patients with blast-phase or accelerated-phase disease.

    In this study, increasing doses of ruxolitinib were given in combination with a standard dose of Decitabine. 21 patients were treated on this study. Importantly, we did not identify a dose of ruxolitinib in combination with Decitabine that was too toxic to be used. The drugs were generally well tolerated when used together. The overall response rate for the trial (a combination of the rate of complete remission rate and partial remission rate) was 57%, which compares favorably with historical outcomes in patients with blast-phase or accelerated-phase disease. Based on these results we have moved forward to a phase II trial combining a fixed dose of ruxolitinib with decitabine, which is currently open in several centers in the US (MPD-RC 109 trial). Click here to read more study details: