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Profile of pomalidomide and its potential in the treatment of myelofibrosis

Authors Gowin K, Mesa R

Received 17 January 2015

Accepted for publication 25 February 2015

Published 2 April 2015 Volume 2015:11 Pages 549—556

DOI https://doi.org/10.2147/TCRM.S69211

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 4

Editor who approved publication: Professor Garry Walsh

Video presented by Ruben A Mesa and Krisstina L Gowin.

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Krisstina L Gowin, Ruben A Mesa

Division of Hematology and Medical Oncology, Mayo Clinic Arizona, Scottsdale, AZ, USA

Abstract: Myelofibrosis, a Philadelphia-negative myeloproliferative neoplasm, is in a new treatment era after the discovery of the JAK2V617F mutation in 2005. JAK inhibitors boast improvements in disease-related symptoms, splenomegaly, and overall survival; however, treatment of myelofibrosis remains a challenge, given the lack of improvement in cytopenias with these agents. Second-generation immunomodulatory agents, such as pomalidomide, have shown efficacy in myelofibrosis-associated anemia within multiple clinical trials. Five major pomalidomide clinical trials have been completed to date, and demonstrate tolerability and efficacy with low-dose pomalidomide (0.5 mg/day) in the treatment of myelofibrosis, and no clinical benefit of elevated dosing regimens (≥2.5 mg/day). Anemia responses ranged from 17% to 36% as per the International Working Group for Myelofibrosis Research and Treatment consensus guidelines, while improvements in splenomegaly were rare, and observed in <1% of most clinical trials. In comparison with earlier immunomodulatory agents, pomalidomide was associated with an improved toxicity profile, with substantially lower rates of myelosuppression and neuropathy. Given the low overall response rate to pomalidomide as a single agent, combination strategies are of particular interest for future studies. Pomalidomide is currently being tested in combination with ruxolitinib, and other novel combinations are likely on the horizon.

Keywords: myelofibrosis, pomalidomide, immunomodulatory, myeloproliferative neoplasm

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