Ruxolitinib for myelofibrosis

Author(s): Clodagh Keohane & Claire Harrison

Myelofibrosis (MF), a myeloproliferative neoplasm, is a disease associated with a significant burden of symptoms, shortened survival and an array of standard treatment regimens which have historically lacked impact and efficacy. The discovery in 2005 of the highly prevalent JAK2V617F-activating tyrosine kinase mutation, strongly associated with myeloproliferative neoplasms, led to the rapid development of a new class of drugs, JAK inhibitors, for the treatment of MF. These drugs have produced a profound effect upon splenomegaly, proliferative blood counts and constitutional symptoms, which are characteristic of MF, and have given hope to both patients and physicians who treat this debilitating disease. This article reviews the current evidence for the use of the JAK inhibitor ruxolitinib, which has completed Phase III trials and with which there is the most extensive clinical experience, as well as assessing other JAK inhibitors in clinical development.