Abstract:
: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 CALR mutation. 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.gov as #NCT01259856.
journal_name
Bloodjournal_title
Bloodauthors
Yacoub A,Mascarenhas J,Kosiorek H,Prchal JT,Berenzon D,Baer MR,Ritchie E,Silver RT,Kessler C,Winton E,Finazzi MC,Rambaldi A,Vannucchi AM,Leibowitz D,Rondelli D,Arcasoy MO,Catchatourian R,Vadakara J,Rosti V,Hexner Edoi
10.1182/blood.2019000428subject
Has Abstractpub_date
2019-10-31 00:00:00pages
1498-1509issue
18eissn
0006-4971issn
1528-0020pii
blood.2019000428journal_volume
134pub_type
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