How I treat polycythemia vera

Blood. 2012 Jul 12;120(2):275-84. doi: 10.1182/blood-2012-02-366054. Epub 2012 May 18.

Abstract

Polycythemia vera (PV) is a clonal disorder characterized by unwarranted production of red blood cells. In the majority of cases, PV is driven by oncogenic mutations that constitutively activate the JAK-STAT signal transduction pathway, such as JAK2 V617F, or exon 12 mutations or LNK mutations. Diagnosis of PV is based on the WHO criteria. Diagnosis of post-PV myelofibrosis is established according to the International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria. Different clinical presentations of PV are discussed. Prognostication of PV is tailored to the most frequent complication during follow-up, namely, thrombosis. Age older than 60 years and prior history of thrombosis are the 2 main risk factors for disease stratification. Correlations are emerging between leukocytosis, JAK2(V617F) mutation, BM fibrosis, and different outcomes of PV, which need to be confirmed in prospective studies. In my practice, hydroxyurea is still the "gold standard" when cytoreduction is needed, even though pegylated IFN-alfa-2a and ruxolitinib might be useful in particular settings. Results of phase 1 or 2 studies concerning these latter agents should however be confirmed by the ongoing randomized phase 3 clinical trials. In this paper, I discuss the main problems encountered in daily clinical practice with PV patients regarding diagnosis, prognostication, and therapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Female
  • Humans
  • Hydroxyurea / therapeutic use
  • Interferon alpha-2
  • Interferon-alpha / therapeutic use
  • Janus Kinase 2 / genetics
  • Janus Kinases / antagonists & inhibitors
  • Male
  • Mutation
  • Nitriles
  • Phlebotomy
  • Platelet Aggregation Inhibitors / therapeutic use
  • Polycythemia Vera / complications
  • Polycythemia Vera / diagnosis
  • Polycythemia Vera / genetics
  • Polycythemia Vera / therapy*
  • Pregnancy
  • Primary Myelofibrosis / diagnosis
  • Primary Myelofibrosis / etiology
  • Prognosis
  • Pyrazoles / therapeutic use
  • Pyrimidines
  • Recombinant Proteins / therapeutic use
  • Risk Factors

Substances

  • Interferon alpha-2
  • Interferon-alpha
  • Nitriles
  • Platelet Aggregation Inhibitors
  • Pyrazoles
  • Pyrimidines
  • Recombinant Proteins
  • ruxolitinib
  • JAK2 protein, human
  • Janus Kinase 2
  • Janus Kinases
  • Hydroxyurea