title: Emerging treatment options for myelofibrosis: focus on pacritinib.
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.
authors.affiliation: Department of Clinical Pharmacy and Pharmaceutical Economics and Policy, University of Southern California, Los Angeles, CA, USA.
authors.forename: Vivian
authors.initials: V
authors.lastname: Chow
authors.affiliation: Department of Pharmacy, University of Southern California Norris Comprehensive Cancer Center, Los Angeles, CA, USA.
authors.forename: Ashley
authors.initials: A
authors.lastname: Weissman
authors.affiliation: Jane 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.
authors.forename: Casey Lee
authors.initials: CL
authors.lastname: O'Connell
authors.affiliation: Mahak Children's Cancer Treatment and Research Center, Tehran, Iran.
authors.forename: Azim
authors.initials: A
authors.lastname: Mehrvar
authors.affiliation: Jane 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.
authors.forename: Mojtaba
authors.initials: M
authors.lastname: Akhtari
keywords: FLT3; JAK2; SB1518; myeloproliferative neoplasms