From the Leukemia Department, The University of Texas M. D. Anderson Cancer Center, Houston, TX
Authors’ disclosures of potential conflicts of interest are found at the end of this article.
Address reprint requests to Srdan Verstovsek, MD, PhD, Associate Professor, Leukemia Department, Box 428, M.D. Anderson Cancer Center, 1515 Holcombe Blvd., Houston, TX, 77030; e-mail:sverstov{at}mdanderson.org
Overview: Significant clinical advances have been made recently in the treatment of myelofibrosis (MF) with the discovery and development of JAK2 inhibitors. These agents show rapid and marked clinical benefits, including reduction of splenomegaly and improvement in constitutional symptoms. Continuing studies with this new class of molecules will allow us to understand their full clinical potential. Data from a study of a new immunomodulatory drug (IMiD) pomalidomide, showed an improvement in anemia, and promising clinical results are emerging from studies of other targeted therapies such as histone deacetylase inhibitors and DNA methyltransferase inhibitors. These advancements are leading the way into a new era of greater understanding of molecular mechanisms of MF and novel therapies that offer patients meaningful improvements in clinical outcomes.
