Polycythemia vera disease burden: contributing factors, impact on quality of life, and emerging treatment options

Brady L. Stein, Alison R. Moliterno, Ramón V. Tiu

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)1965-1976
Number of pages12
JournalAnnals of hematology
Issue number12
StatePublished - Dec 2014


  • Burden of disease
  • JAK2
  • Myeloproliferative neoplasms
  • Polycythemia vera
  • Quality of life
  • Symptoms

ASJC Scopus subject areas

  • Hematology


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