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Patients with polycythemia vera (PV), a chronic myeloproliferative disorder, are generally treated with phlebotomy to maintain hematocrit values of <45%. Those who are unable to tolerate phlebotomy or who have progressive splenomegaly or thromboembolic events are prescribed hydroxyurea. Patients refractory to hydroxyurea are given other drugs, which are of limited efficacy and safety.
Now, investigators have conducted an industry-sponsored, multicenter, randomized, open-label, phase III trial (RESPONSE) of ruxolitinib, a Janus kinase inhibitor that was shown to be effective in early-phase trials of patients with PV. A total of 222 PV patients with resistance or intolerance to hydroxyurea were randomized to receive ruxolitinib or standard the…