Testing the Effect of Taking Ruxolitinib and CPX-351 in Combination for the Treatment of Advanced Phase Myeloproliferative Neoplasms
Key claim: A phase I/II trial is evaluating the optimal dose and efficacy of ruxolitinib combined with CPX-351 (liposomal daunorubicin/cytarabine) in patients with accelerated or blast phase myeloproliferative neoplasms.
Abstract
This phase I/II trial studies the best dose of ruxolitinib when given together with CPX-351 and to see how well they work in treating patients with accelerated phase or blast phase myeloproliferative neoplasm. Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. CPX-351 is a mixture of 2 chemotherapy drugs (daunorubicin and cytarabine) given for leukemia in small fat-based particles (liposomes) to improve the drug getting into cancer cells. Giving ruxolitinib and CPX-351 may work better in treating patients with secondary acute myeloid leukemia compared to CPX-351 alone.
Why this matters
Accelerated and blast-phase MPN — most often arising from PMF — carries dismal prognosis with no standard of care, and AML-style induction has historically performed poorly. Layering ruxolitinib onto CPX-351, a liposomal daunorubicin/cytarabine formulation with proven benefit in secondary AML, tests whether continued JAK-STAT suppression plus an MPN-biology-aware chemotherapy backbone can improve transformation outcomes. This is the first formal phase I/II evaluation of this specific combination in advanced-phase MPN.