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Author Notes:

John Mascarenhas, Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1079, New York, NY 10029; e-mail: john.mascarenhas@mssm.edu

J.M., H.E.K., J.T.P., A.R., C.N.H., M.F.M., A.M.V., J.E., J.-J.K., R.T.S., R.S.W., L.P., J.D.G., T.B., R.M., G.T., R.A.M., A.C.D., and R.H. contributed to the design of the clinical trial; J.M., H.E.K., A.R., D.B., A.Y., C.N.H., M.F.M., A.M.V., J.E., C.L.O., J.-J.K., A.J.M., E.F.W., D.S.L., V.D.S., M.O.A., C.M.K., R.C., D.R., R.T.S., A.B., A.N., M.K., M.E.S., V.N., N.F., R.S.W., L.P., J.D.G., T.B., R.M., G.T., R.K.R., R.A.M., A.C.D., and R.H. wrote the manuscript; J.M., H.E.K., J.T.P., A.R., D.B., A.Y., C.N.H., M.F.M., A.M.V., J.E., C.L.O., J.-J.K., A.J.M., E.F.W., D.S.L., V.D.S., M.O.A., C.M.K., R.C., D.R., R.T.S., A.B., A.N., M.K., M.F.L., J.E.A.O., E.M., L.S., M.E.S., V.N., J.T., N.F., A.V.P., R.S.W., L.P., J.D.G., T.B., R.M., G.T., R.K.R., R.A.M., A.C.D., and R.H. contributed to patient recruitment, data collection, and analysis; H.E.K., L.P., J.D.G., and A.C.D. performed statistical analyses; and all authors gave final approval of the version to be published and agreed to be accountable for questions related to the accuracy and integrity of the work.

J.M. reports clinical trial research support paid to the institution from Incyte, Roche, Novartis, CTI Biopharma, Janssen Pharmaceuticals, Geron, Kartos, AbbVie, Merck, Promedior, PharmaEssentia, and Celgene/BMS and is a clinical trial steering committee member, scientific advisory board member, and consultant for Roche, CTI Biopharma, Constellation, Novartis, Galecto, PharmaEssentia, Sierra Oncology, AbbVie, Kartos, Incyte, and Celgene/BMS. reports clinical trial research support paid to the institution from AbbVie, AI, Astellas, Forma, Incyte, Kite, and Takeda. E.F.W. reports advisory boards for Incyte and Gilead Sciences. A.Y. reports consultation and speaker honoraria for Incyte, Seattle Genetics, and Novartis. A.R. reports consultation and speaker honoraria for Novartis, Amgen, Roche, Celgene, and Italfarmaco. A.M.V. reports speaker honoraria from Novartis and Celgene and fees for participation on advisory boards for Novartis, CTI, and Celgene. D.R. reports consulting honoraria from Incyte. M.O.A. reports research grant support paid to institution from Incyte, CTI Biopharma, Samus Therapeutics, Janssen Pharmaceuticals, and Gilead. R.T.S. reports consultancy and speaker bureau fees from Pharmaessentia. R.K.R. has received consulting fees from Stemline Corporation, Celgene, Agios Pharmaceuticals, Apexx Oncology, Beyond Spring, Partner Therapeutics, and Jazz Pharmaceuticals and has received research funding from Constellation Pharmaceuticals, Incyte, and Stemline Therapeutics. R.M. reports research support from Incyte, Genentech, CTI, Promedior, and AbbVie and is a consultant for Novartis, Sierra Oncology, and La Jolla Pharma. R.H. reports research support from Roche. The remaining authors declare no competing financial interests.

Subjects:

Research Funding:

This work was supported by a grant from the National Cancer Institute, National Institutes of Health (MPN Research Consortium, 5P01CA108671-09), a Cancer Center Support Grant/Core grant to Memorial Sloan-Kettering Cancer Center (P30 CA008748), and generous independent, unrestricted support from Roche Genentech. R.K.R. is supported by the National Cancer Institute, National Institutes of Health (1K08CA188529-01).

Keywords:

  • Science & Technology
  • Life Sciences & Biomedicine
  • Hematology
  • NEOPLASMS
  • GROWTH

A randomized phase 3 trial of interferon-alpha vs hydroxyurea in polycythemia vera and essential thrombocythemia

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Journal Title:

BLOOD

Volume:

Volume 139, Number 19

Publisher:

, Pages 2931-2941

Type of Work:

Article | Final Publisher PDF

Abstract:

The goal of therapy for patients with essential thrombocythemia (ET) and polycythemia vera (PV) is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for patients with ET and PV at high risk for vascular complications. Myeloproliferative Disorders Research Consortium 112 was an investigator-initiated, phase 3 trial comparing HU to pegylated IFN-α (PEG) in treatment-naïve, high-risk patients with ET/PV. The primary endpoint was complete response (CR) rate at 12 months. A total of 168 patients were treated for a median of 81.0 weeks. CR for HU was 37% and 35% for PEG (P = .80) at 12 months. At 24 to 36 months, CR was 20% to 17% for HU and 29% to 33% for PEG. PEG led to a greater reduction in JAK2V617F at 24 months, but histopathologic responses were more frequent with HU. Thrombotic events and disease progression were infrequent in both arms, whereas grade 3/4 adverse events were more frequent with PEG (46% vs 28%). At 12 months of treatment, there was no significant difference in CR rates between HU and PEG. This study indicates that PEG and HU are both effective treatments for PV and ET. With longer treatment, PEG was more effective in normalizing blood counts and reducing driver mutation burden, whereas HU produced more histopathologic responses. Despite these differences, both agents did not differ in limiting thrombotic events and disease progression in high-risk patients with ET/PV. This trial was registered at www.clinicaltrials.gov as #NCT01259856.

Copyright information:

© 2022 by The American Society of Hematology.

This is an Open Access work distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (https://creativecommons.org/licenses/by-nc-nd/4.0/rdf).
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