title: Polycythemia vera disease burden: contributing factors, impact on quality of life, and emerging treatment options.
abstract: Polycythemia vera (PV) is a chronic myeloproliferative neoplasm characterized by clonal expansion of a hematopoietic progenitor, erythrocytosis, often leukocytosis and/or thrombocytosis, and nearly always an activating mutation in Janus kinase 2 (JAK2). The PV symptom burden can be considerable, in part driven by small or large vessel thrombotic tendency, splenomegaly, fatigue, pruritus, and a chronic risk of disease transformation to myelofibrosis or acute myeloid leukemia. In addition, patients with PV have an increased risk of mortality compared with the general population that often results from cardiovascular complications or disease transformation. Further, healthcare utilization and costs are higher in patients with PV than noncancer controls. First-line therapy options for high-risk patients may effectively manage PV in some instances; however, some patients do not receive adequate benefit from current treatment options and experience a more severe disease burden as a result. This may be especially true for those patients who are resistant to or intolerant of hydroxyurea or interferon-based therapies. New treatments currently being investigated in phase 3 clinical trials may alleviate disease burden in this patient population.
authors.affiliation: Department of Medicine, Division of Hematology and Oncology, Northwestern University Feinberg School of Medicine, 645 N. Michigan Avenue, Suite 1020, Chicago, IL, 60611, USA, bstein@nmff.org.
authors.forename: Brady L
authors.initials: BL
authors.lastname: Stein
authors.forename: Alison R
authors.initials: AR
authors.lastname: Moliterno
authors.forename: Ramón V
authors.initials: RV
authors.lastname: Tiu
mesh_terms: D017326:Clinical Trials, Phase III as Topic; D003131:Combined Modality Therapy; D017281:Cost of Illness; D018450:Disease Progression; D005221:Fatigue / Q000209:etiology; D017048:Health Care Costs; D006474:Hemorrhagic Disorders / Q000209:etiology; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007372:Interferons / Q000627:therapeutic use; D053614:Janus Kinase 2 / Q000235:genetics; D015470:Leukemia, Myeloid, Acute / Q000209:etiology; D020125:Mutation, Missense; D016609:Neoplasms, Second Primary / Q000209:etiology / Q000401:mortality; D009570:Nitriles; D010885:Pipobroman / Q000627:therapeutic use; D011087:Polycythemia Vera / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy / Q000191:economics / Q000453:epidemiology / Q000235:genetics; D055728:Primary Myelofibrosis / Q000209:etiology; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D011720:Pyrazoles / Q000627:therapeutic use; D011743:Pyrimidines; D011788:Quality of Life; D035703:Therapies, Investigational; D019851:Thrombophilia / Q000209:etiology
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; D010885:Pipobroman; C540383:ruxolitinib; D007372:Interferons; C507924:JAK2 protein, human; D053614:Janus Kinase 2; D006918:Hydroxyurea