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

Autor: John Mascarenhas, Heidi E. Kosiorek, Josef T. Prchal, Alessandro Rambaldi, Dmitriy Berenzon, Abdulraheem Yacoub, Claire N. Harrison, Mary Frances McMullin, Alessandro M. Vannucchi, Joanne Ewing, Casey L. O'Connell, Jean-Jacques Kiladjian, Adam J. Mead, Elliott F. Winton, David S. Leibowitz, Valerio De Stefano, Murat O. Arcasoy, Craig M. Kessler, Rosalind Catchatourian, Damiano Rondelli, Richard T. Silver, Andrea Bacigalupo, Arnon Nagler, Marina Kremyanskaya, Max F. Levine, Juan E. Arango Ossa, Erin McGovern, Lonette Sandy, Mohamad E. Salama, Vesna Najfeld, Joseph Tripodi, Noushin Farnoud, Alexander V. Penson, Rona Singer Weinberg, Leah Price, Judith D. Goldberg, Tiziano Barbui, Roberto Marchioli, Gianni Tognoni, Raajit K. Rampal, Ruben A. Mesa, Amylou C. Dueck, Ronald Hoffman
Rok vydání: 2021
Předmět:
Zdroj: Mascarenhas, J, Kosiorek, H E, Prchal, J T, Rambaldi, A, Berenzon, D, Yacoub, A, Harrison, C N, McMullin, M F F, Vannucchi, A M, Ewing, J, O'Connell, C L, Kiladjian, J-J, Mead, A J, Winton, E F, Leibowitz, D S, De Stefano, V, Arcasoy, M O, Kessler, C M, Catchatourian, R, Rondelli, D, Silver, R T, Bacigalupo, A, Nagler, A, Kremyanskaya, M, Levine, M F, Arango Ossa, J E, McGovern, E M, Sandy, L, Salama, M E, Najfeld, V, Tripodi, J, Farnoud, N, Penson, A V, Weinberg, R S, Price, L, Goldberg, J D, Barbui, T, Marchioli, R, Tognoni, G, Rampal, R K, Mesa, R A, Dueck, A C & Hoffman, R 2022, ' A Randomized, Phase 3, Trial of Interferon-α versus Hydroxyurea in Polycythemia Vera and Essential Thrombocythemia ', Blood . https://doi.org/10.1182/blood.2021012743
ISSN: 1528-0020
DOI: 10.1182/blood.2021012743
Popis: The goal of therapy for essential thrombocythemia (ET) and polycythemia vera (PV) patients is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for ET and PV patients 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 ET/PV patients. 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=0.80) at 12 months. At 24/36 months, CR was 20%/17% for HU and 29%/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, while 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, while HU produced more histopathologic responses. Despite these differences, both agents did not differ in limiting thrombotic events and disease progression in high-risk ET/PV patients. (Funded by the National Cancer Institute, 5P01CA108671-09; clinicaltrials.gov number (NCT01259856). [Abstract copyright: Copyright © 2022 American Society of Hematology.]
Databáze: OpenAIRE
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