Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea

Abdulraheem Yacoub, John Mascarenhas, Heidi Kosiorek, Josef T. Prchal, Dmitry Berenzon, Maria R. Baer, Ellen Ritchie, Richard T. Silver, Craig Kessler, Elliott Winton, Maria Chiara Finazzi, Alessandro Rambaldi, Alessandro M. Vannucchi, David Leibowitz, Damiano Rondelli, Murat O. Arcasoy, Rosalind Catchatourian, Joseph Vadakara, Vittorio Rosti, Elizabeth HexnerMarina Kremyanskaya, Lonette Sandy, Joseph Tripodi, Vesna Najfeld, Noushin Farnoud, Elli Papaemmanuil, Mohamed Salama, Rona Singer-Weinberg, Raajit Rampal, Judith D. Goldberg, Tiziano Barbui, Ruben Mesa, Amylou C. Dueck, Ronald Hoffman

Research output: Contribution to journalArticle

18 Scopus citations

Abstract

Prior studies have reported high response rates with recombinant interferon-a (rIFN-a) therapy in patients with essential thrombocythemia (ET) and polycythemia vera (PV). To further define the role of rIFN-a,we investigated the outcomes of pegylated-rIFN-a2a (PEG) therapy in ET and PV patients previously treated with hydroxyurea (HU). The Myeloproliferative Disorders Research Consortium (MPD-RC)-111 study was an investigator-initiated, international, multicenter, phase 2 trial evaluating the ability of PEG therapy to induce complete (CR) and partial (PR) hematologic responses in patients with high-risk ET or PVwho were either refractory or intolerant to HU. The study included 65 patients with ET and 50 patients with PV. The overall response rates (ORRs; CR/PR) at 12 monthswere 69.2%(43.1% and 26.2%) in ET patients and 60% (22% and 38%) in PV patients. CR rates were higher in CALR-mutated ET patients (56.5% vs 28.0%; P 5 .01), compared with those in subjects lacking a CALR mutation. The median absolute reduction in JAK2V617F variant allele fraction was 26% (range, 284%to 47%) in patients achieving a CR vs 14%(range, 218% to 56%) in patients with PR or nonresponse (NR). Therapy was associated with a significant rate of adverse events (AEs); most were manageable, and PEG discontinuation related to AEs occurred in only 13.9% of subjects. We conclude that PEG is an effective therapy for patients with ET or PV who were previously refractory and/or intolerant of HU.

Original languageEnglish (US)
Pages (from-to)1498-1509
Number of pages12
JournalBlood
Volume134
Issue number18
DOIs
StatePublished - Oct 31 2019

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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    Yacoub, A., Mascarenhas, J., Kosiorek, H., Prchal, J. T., Berenzon, D., Baer, M. R., Ritchie, E., Silver, R. T., Kessler, C., Winton, E., Finazzi, M. C., Rambaldi, A., Vannucchi, A. M., Leibowitz, D., Rondelli, D., Arcasoy, M. O., Catchatourian, R., Vadakara, J., Rosti, V., ... Hoffman, R. (2019). Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea. Blood, 134(18), 1498-1509. https://doi.org/10.1182/blood.2019000428