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Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.

N Engl J Med

February 2024

From the Division of Hematology and Medical Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai (M.K., Y.Z.G., R.H.), and the Division of Hematology and Medical Oncology, Weill Cornell Medical College (E.K.R.) - both in New York; the Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL (A.T.K.); the Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston (N.P., S.V.); the Division of Hematology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford (J.G.), and Clinical Research and Development, Protagonist Therapeutics, Newark (A.M., S.R.S, N.B.M., F.H.V., S.K., S.G.) - both in California; the Department of Hematology and Medical Oncology, Cleveland Clinic Taussig Cancer Institute, Cleveland (A.G.); the Mayo Clinic, Phoenix, AZ (J.P.); the Rogel Cancer Center, University of Michigan Health, Ann Arbor (K.P.); All India Institute of Medical Sciences, Rishikesh, India (U.K.N.); and the University of Kansas Cancer Center, Westwood (A.Y.).

Article Synopsis
  • Polycythemia vera is a chronic blood disorder leading to high red blood cell counts, and rusfertide is a treatment that mimics a hormone controlling iron levels, but its safety and effectiveness in these patients were previously unknown.
  • The REVIVE trial tested rusfertide in two parts: a 28-week dose-finding phase involving 70 patients, followed by a 12-week phase where participants received either rusfertide or a placebo.
  • Results showed that patients treated with rusfertide had significantly fewer annual phlebotomies (0.6 vs. 8.7) and a better response rate (60% vs. 17%) compared to the placebo group, along with improvements
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