title: Assessing efficacy in myelofibrosis treatment: a focus on JAK inhibition.
abstract: Myelofibrosis (MF) is characterized by splenomegaly, anemia and a debilitating symptom burden (e.g., fatigue, night sweats, pruritus, bone and muscle pain, undesired weight loss). Moreover, these symptoms impair activities of daily living and quality of life. Until recently, there have been no approved therapies for MF, and management of MF has been predominantly palliative. Dysregulated JAK-STAT signaling is associated with the pathologic MF disease state. A novel class of therapies, the JAK inhibitors, offers the potential to abrogate this pathologic signaling pathway. In clinical trials of patients with intermediate- and high-risk MF, JAK inhibitors have demonstrated efficacy in reducing splenomegaly and MF-associated symptoms. Evidence from ruxolitinib trials also suggests that JAK inhibitors may improve survival outcomes.
authors.affiliation: H Lee Moffitt Cancer Center and Research Institute, 12902 Magnolia Drive, Tampa, FL 33612, USA.
authors.forename: Rami
authors.initials: R
authors.lastname: Komrokji
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D000818:Animals; D006801:Humans; D053612:Janus Kinases / Q000037:antagonists & inhibitors* / Q000378:metabolism; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000201:enzymology*; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D015398:Signal Transduction
chemical_list: D047428:Protein Kinase Inhibitors; D053612:Janus Kinases