TY - JOUR
T1 - Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea
AU - Yacoub, Abdulraheem
AU - Mascarenhas, John
AU - Kosiorek, Heidi
AU - Prchal, Josef T.
AU - Berenzon, Dmitry
AU - Baer, Maria R.
AU - Ritchie, Ellen
AU - Silver, Richard T.
AU - Kessler, Craig
AU - Winton, Elliott
AU - Finazzi, Maria Chiara
AU - Rambaldi, Alessandro
AU - Vannucchi, Alessandro M.
AU - Leibowitz, David
AU - Rondelli, Damiano
AU - Arcasoy, Murat O.
AU - Catchatourian, Rosalind
AU - Vadakara, Joseph
AU - Rosti, Vittorio
AU - Hexner, Elizabeth
AU - Kremyanskaya, Marina
AU - Sandy, Lonette
AU - Tripodi, Joseph
AU - Najfeld, Vesna
AU - Farnoud, Noushin
AU - Papaemmanuil, Elli
AU - Salama, Mohamed
AU - Singer-Weinberg, Rona
AU - Rampal, Raajit
AU - Goldberg, Judith D.
AU - Barbui, Tiziano
AU - Mesa, Ruben
AU - Dueck, Amylou C.
AU - Hoffman, Ronald
N1 - Publisher Copyright:
© 2019 by The American Society of Hematology.
PY - 2019/10/31
Y1 - 2019/10/31
N2 - 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.
AB - 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.
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U2 - 10.1182/blood.2019000428
DO - 10.1182/blood.2019000428
M3 - Article
C2 - 31515250
AN - SCOPUS:85074499705
SN - 0006-4971
VL - 134
SP - 1498
EP - 1509
JO - Blood
JF - Blood
IS - 18
ER -