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

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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.

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Acknowledgments

Writing assistance was provided by Cory Pfeiffenberger, Ph.D. (Complete Healthcare Communications, Inc.), whose work was funded by Incyte Corporation.

Financial and competing interests disclosure

Brady L. Stein has participated in speaker’s bureau for Incyte Corporation and advisory meetings for Sanofi Oncology and Incyte Corporation. Ramón V. Tiu has participated in speaker’s bureaus for Incyte Corporation and Novartis and advisory meetings for Gilead, Incyte Corporation, and Novartis. Alison R. Moliterno has participated in advisory boards for Incyte Corporation.

Conflict of interest

The authors declare that they have no conflict of interest.

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Authors and Affiliations

  1. Department of Medicine, Division of Hematology and Oncology, Northwestern University Feinberg School of Medicine, 645 N. Michigan Avenue, Suite 1020, Chicago, IL, 60611, USA

    Brady L. Stein

  2. Johns Hopkins University School of Medicine, Baltimore, MD, USA

    Alison R. Moliterno

  3. Cleveland Clinic Taussig Cancer Institute, Cleveland, OH, USA

    Ramón V. Tiu

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  1. Brady L. Stein
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  2. Alison R. Moliterno
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  3. Ramón V. Tiu
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Correspondence to Brady L. Stein.

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Stein, B.L., Moliterno, A.R. & Tiu, R.V. Polycythemia vera disease burden: contributing factors, impact on quality of life, and emerging treatment options. Ann Hematol 93, 1965–1976 (2014). https://doi.org/10.1007/s00277-014-2205-y

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