title: Novel and emerging therapies for the treatment of polycythemia vera.
abstract: Polycythemia vera (PV) is a chronic myeloproliferative neoplasm defined by erythrocytosis and often accompanied by leukocytosis and thrombocytosis. Current treatment options, including IFN-α and hydroxyurea, effectively manage PV in many patients. However, some high-risk patients, particularly those who become hydroxyurea-intolerant/resistant, may benefit from IFN-α or new treatment options. A better understanding of PV pathophysiology, including the role of the JAK/STAT pathway, has inspired the development of new therapies. Several JAK inhibitors directly target JAK/STAT pathway activation and have been evaluated in Phase II/III trials with promising results. Pegylated variants of IFN-α, which reduce dosing frequency and toxicity associated with recombinant IFN-α, have yielded favorable efficacy results in Phase II trials. Finally, histone deacetylase inhibitors have been developed to manage PV at the level of chromatin-regulated gene expression. The earliest Phase III results from these next-generation therapies are expected in 2014.
authors.affiliation: Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd, Unit 418, Houston, TX 77030, USA.
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
authors.forename: Rami S
authors.initials: RS
authors.lastname: Komrokji
mesh_terms: D006801:Humans; D011087:Polycythemia Vera / Q000188:drug therapy*
keywords: IFN-α; JAK2; MPN; interferon-α; myeloproliferative neoplasms; polycythemia vera; ruxolitinib