Ruxolitinib for the treatment of patients with polycythemia vera

• Type of Material: Article
• Authors: Jean-Jacques Kiladjian, Hôpital Saint-Louis & Université Paris Diderot; Elliott Winton, Emory University; Moshe Talpaz, University of Michigan; Srdan Verstovsek, The University of Texas
• Language: English
• Date: 2015-05-17
• Publisher: Expert Reviews (formerly Future Drugs)
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © Informa UK, Ltd.
• Final Published Version (URL): http://dx.doi.org/10.1586/17474086.2015.1045869
• Title of Journal or Parent Work: Expert Review of Hematology
• ISSN: 1747-4086
• Volume: 8
• Issue: 4
• Start Page: 391
• End Page: 401
• Grant/Funding Information: SV has received research funding from Astrazeneca, Bristol Myers Squibb, Celgene, Cell Therapeutics Inc., Galena BioPharma, Geron, Gilead Sciences Inc., Incyte Corporation, Infinity Pharmaceuticals, Lilly Oncology, NS Pharma, Pfizer, Promedior, Roche, and Seattle Genetics.; EFW has received research funding from Gilead Sciences Inc., Incyte Corporation, Pfizer, and Sanofi, and has served on advisory boards for Incyte Corporation.; J-JK has received honoraria and research funding from Novartis Pharmaceuticals and has served on advisory boards for Novartis Pharmaceuticals.; MT has received research funding from ARIAD, Bristol-Myers Squibb, Sanofi, Incyte Corporation, and Pfizer.
• Keywords: QUALITY-OF-LIFE; INCB018424 PHOSPHATE; CHRONIC MYELOPROLIFERATIVE NEOPLASMS; Janus kinase 1; ESSENTIAL THROMBOCYTHEMIA; SIGNAL-TRANSDUCTION; Life Sciences & Biomedicine; myeloproliferative neoplasm; erythrocytosis; thrombocytosis; Hematology; HEALTHY-VOLUNTEERS; ruxolitinib; PEGYLATED INTERFERON ALPHA-2A; TYROSINE KINASE JAK2; Science & Technology; splenomegaly; Janus kinase 2; polycythemia vera; RANDOMIZED-TRIAL; PRECLINICAL CHARACTERIZATION
• Research Categories: Health Sciences, Pathology; Health Sciences, Oncology; Health Sciences, Medicine and Surgery

Abstract

Polycythemia vera (PV) is a hematopoietic proliferative disorder associated with Janus-associated kinase/signal transducer and activator of transcription pathway dysregulation resulting in erythrocytosis and, possibly, leukocytosis and thrombocytosis. Patients diagnosed with PV experience a broad range of symptoms associated with a reduced quality of life, often develop splenomegaly, and have an increased risk of death compared with age-matched subjects without PV. Current treatment options, notably hydroxyurea, help with disease management; however, insufficient efficacy or progressive resistance occurs in some patients, highlighting the need for new treatment options. Ruxolitinib is an oral JAK1/JAK2 inhibitor that has been evaluated in Phase II and III clinical trials in patients with PV, who are intolerant of or resistant to hydroxyurea. In this setting, ruxolitinib treatment has demonstrated normalization of blood cell counts, reduction in splenomegaly and improvements in PV-related symptom burden.