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Blood. 2019 Oct 31;134(18):1498-1509. doi: 10.1182/blood.2019000428.

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

Author information

1
Division of Hematologic Malignancies and Cellular Therapeutics, The University of Kansas Cancer Center, Westwood, KS.
2
Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
3
Mayo Clinic, Scottsdale, AZ.
4
Division of Hematology, University of Utah School of Medicine and.
5
Huntsman Cancer Center, Salt Lake City, UT.
6
Comprehensive Cancer Center, Wake Forest University Medical Center, Wake Forest Health, Winston-Salem, NC.
7
Greenebaum Comprehensive Cancer Center, University of Maryland, Baltimore, MD.
8
Division of Hematology and Oncology, Department of Medicine, Richard T. Silver Myeloproliferative Neoplasms Center, Weill Cornell Medical College, New York, NY.
9
Georgetown University Medical Center, Washington, DC.
10
Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA.
11
Department of Hematology, Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Bergamo, Italy.
12
Department of Oncology, University of Milan, Ospedale Papa Giovanni XXIII, Bergamo, Italy.
13
Center for Research and Innovation of Myeloproliferative Neoplasms, Azienda Ospedaliero-Universitaria Careggi, and.
14
Denothe Excellence Center, University of Florence, Florence, Italy.
15
Department of Oncology, Palo Alto Medical Foundation, Sutter Health, Palo Alto, CA.
16
Division of Hematology and Oncology, University of Illinois, Chicago, IL.
17
Division of Hematology, Duke University School of Medicine, Durham, NC.
18
Department of Oncology, John H. Stroger Jr. Hospital of Cook County, Chicago, IL.
19
Geisinger Medical Center, Danville, PA.
20
Laboratory of Biochemistry, Biotechnology, and Advanced Diagnosis, Center for the Study of Myelofibrosis, Istituto Di Ricovero e Cura a Carattere Scientifico, Foundation Policlinico San Matteo, Pavia, Italy.
21
Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
22
Department of Pathology and.
23
Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY.
24
Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY.
25
Computational Oncology.
26
Center for Hematological Malignancies, and.
27
Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY.
28
Mayo Clinic Laboratories, Rochester, MN.
29
New York Blood Center, New York, NY.
30
Department of Population Health and.
31
Department of Environmental Medicine, New York University School of Medicine, New York, NY.
32
Ospedali Riuniti, Bergamo, Italy; and.
33
UT Health San Antonio Cancer Center, San Antonio, TX.

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.

Comment in

PMID:
31515250
PMCID:
PMC6839950
DOI:
10.1182/blood.2019000428
[Indexed for MEDLINE]
Free PMC Article

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