Journal
BLOOD
Volume 134, Issue 18, Pages 1498-1509Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood.2019000428
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Funding
- National Cancer Institute (NCI), National Institutes of Health (MPN Research Consortium) [5P01CA108671-09]
- NCI Cancer Center Support Grant/Core Grant [P30CA008748]
- Roche Genentech
- NCI [1K08CA188529-01]
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Prior studies have reported high response rates with recombinant interferon-alpha (rIFN-alpha) therapy in patients with essential thrombocythemia (ET) and polycythemia vera (PV). To further define the role of rIFN-alpha, we investigated the outcomes of pegylated-rIFN-alpha 2a (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.
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