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


Authors: 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.; Hexner, E.; Kremyanskaya, M.; Sandy, L.; Tripodi, J.; Najfeld, V.; Farnoud, N.; Papaemmanuil, E.; Salama, M.; Singer-Weinberg, R.; Rampal, R.; Goldberg, J. D.; Barbui, T.; Mesa, R.; Dueck, A. C.; Hoffman, R.
Article Title: Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea
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. © 2019 by The American Society of Hematology.
Keywords: adult; treatment outcome; middle aged; major clinical study; hydroxyurea; janus kinase 2; constipation; fatigue; neutropenia; diarrhea; drug withdrawal; side effect; conference paper; prospective study; allele; edema; pain; phase 2 clinical trial; anemia; leukopenia; nausea; thrombocytopenia; vomiting; myalgia; genetic variation; creatinine; bone pain; creatinine blood level; abdominal pain; alanine aminotransferase blood level; arthralgia; coughing; dizziness; dyspnea; hyperuricemia; injection site reaction; lymphocytopenia; pruritus; rash; alanine aminotransferase; insomnia; depression; multicenter study; urinary tract infection; flu like syndrome; headache; drug dose titration; polycythemia vera; thrombocythemia; upper respiratory tract infection; muscle cramp; peginterferon alpha2a; human; male; female; priority journal
Journal Title: Blood
Volume: 134
Issue: 18
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2019-10-31
Start Page: 1498
End Page: 1509
Language: English
DOI: 10.1182/blood.2019000428
PUBMED: 31515250
PROVIDER: scopus
DOI/URL:
Notes: Source: Scopus
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MSK Authors
  1. Raajit Kumar Rampal
    150 Rampal