دورية
Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea
| العنوان: | Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea |
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| المؤلفون: | Yacoub, Abdulraheem, Mascarenhas, John, Kosiorek, Heidi, Prchal, Josef T., Berenzon, Dmitry, Baer, Maria R., Ritchie, Ellen, Silver, Richard T., Kessler, Craig, Winton, Elliott, Finazzi, Maria Chiara, Rambaldi, Alessandro, Vannucchi, Alessandro M., Leibowitz, David, Rondelli, Damiano, Arcasoy, Murat O., Catchatourian, Rosalind, Vadakara, Joseph, Rosti, Vittorio, Hexner, Elizabeth, Kremyanskaya, Marina, Sandy, Lonette, Tripodi, Joseph, Najfeld, Vesna, Farnoud, Noushin, Papaemmanuil, Elli, Salama, Mohamed, Singer-Weinberg, Rona, Rampal, Raajit, Goldberg, Judith D., Barbui, Tiziano, Mesa, Ruben, Dueck, Amylou C., Hoffman, Ronald |
| المصدر: | Blood; October 2019, Vol. 134 Issue: 18 p1498-1509, 12p |
| مستخلص: | Prior studies have reported high response rates with recombinant interferon-α (rIFN-α) therapy in patients with essential thrombocythemia (ET) and polycythemia vera (PV). To further define the role of rIFN-α, we investigated the outcomes of pegylated-rIFN-α2a (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 PV who 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 months were 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= .01), compared with those in subjects lacking a CALRmutation. The median absolute reduction in JAK2V617F variant allele fraction was −6% (range, −84% to 47%) in patients achieving a CR vs +4% (range, −18% 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. This trial was registered at www.clinicaltrials.govas #NCT01259856. |
| قاعدة البيانات: | Supplemental Index |
Full Text Finder | تدمد: | 00064971 15280020 |
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| DOI: | 10.1182/blood.2019000428 |