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Treatment for polycythemia vera is based on phlebotomy and induction of iron deficiency to maintain hematocrit <45% and aspirin to reduce risk for thromboembolic events; many patients supplement with cytoreductive hydroxyurea, JAK2 inhibitors, or interferon alfa. In an industry-funded phase 2 trial with a novel design, investigators tested the efficacy and safety of rusfertide, a peptide mimetic of endogenous hepcidin that induces functional iron deficiency and decreases erythropoiesis.
In part 1 of the study, 70 patients with phlebotomy-dependent disease received a 28-week dose-finding course of weekly subcutaneous rusfertide administered at 10 to 120 mg, dosed to maintain hematocrit <45%. During part 2, 59 patients were randomized to a 12-…