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Janus kinase (JAK), which mediates signal transduction for myeloid proliferation and cytokine expression, is dysregulated in myelofibrosis. A prior phase I–II trial demonstrated that, for patients with myelofibrosis, ruxolitinib (formerly INCB018424), an orally administered inhibitor of JAK1 and JAK2, provided impressive responses with few serious adverse effects (JW Oncol Hematol Sep 15 2010). To further evaluate ruxolitinib in this setting, investigators conducted two industry-sponsored, randomized, phase III trials.
In the study by Harrison and colleagues, 219 myelofibrosis patients received ruxolitinib twice daily according to platelet count (15 mg for 55 patients with counts ≤200,000/µL or 20 mg for 91 patients with counts >200,000/µL) …