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Emerging treatment options for myelofibrosis: focus on pacritinib

Authors Chow V, Weissman A, O'Connell C, Mehrvar A, Akhtari M

Received 6 August 2015

Accepted for publication 9 January 2016

Published 4 May 2016 Volume 2016:9 Pages 2655—2665


Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Professor Da Li

Peer reviewer comments 3

Editor who approved publication: Dr Faris Farassati

Vivian Chow,1 Ashley Weissman,2 Casey Lee O’Connell,3 Azim Mehrvar,4 Mojtaba Akhtari3

1Department of Clinical Pharmacy and Pharmaceutical Economics and Policy, University of Southern California, 2Department of Pharmacy, 3Jane Anne Nohl Division of Hematology and Center for the Study of Blood Diseases, University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA, USA; 4Mahak Children’s Cancer Treatment and Research Center, Tehran, Iran

Abstract: Myelofibrosis (MF) is a myeloid malignancy associated with a heavy symptomatic burden that decreases quality of life and presents a risk for leukemic transformation. While there are limited curative treatments, the recent discovery of the Janus kinase/signal transducer and activator of transcription (JAK/STAT) pathway dysregulation has led to many clinical investigations for new treatment approaches. This review provides practical knowledge on the disease state, an overview of treatment options, and specifically focuses on the efficacy and safety of pacritinib in the management of MF. Pacritinib is a novel selective inhibitor of JAK2 and FMS-related tyrosine kinase 3 (FLT3) currently in Phase III trials for the treatment of MF. Thus far, studies have demonstrated clinical efficacy in reducing splenomegaly and constitutional symptoms. Common adverse events were gastrointestinal in nature, while hematologic toxicity was limited. However, it was announced that all ongoing clinical trials on pacritinib have been placed on hold by the US Food and Drug Administration in February 2016, due to concerns for increased intracranial hemorrhage and cardiac events. With comprehensive risk-benefit analysis of clinical trial data, the utility of pacritinib in the management of MF may be more clearly defined.

JAK2, FLT3, myeloproliferative neoplasms, SB1518

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