Current and future treatment options for polycythemia vera

Ann Hematol. 2015 Jun;94(6):901-10. doi: 10.1007/s00277-015-2357-4. Epub 2015 Apr 2.

Abstract

Patients with polycythemia vera (PV), a myeloproliferative neoplasm characterized by an elevated red blood cell mass, are at high risk of vascular and thrombotic complications and have reduced quality of life due to a substantial symptom burden that includes pruritus, fatigue, constitutional symptoms, microvascular disturbances, and bleeding. Conventional therapeutic options aim at reducing vascular and thrombotic risk, with low-dose aspirin and phlebotomy as first-line recommendations for patients at low risk of thrombotic events and cytoreductive therapy (usually hydroxyurea or interferon alpha) recommended for high-risk patients. However, long-term effective and well-tolerated treatments are still lacking. The discovery of mutations in Janus kinase 2 (JAK2) as the underlying molecular basis of PV has led to the development of several targeted therapies, including JAK inhibitors, and results from the first phase 3 clinical trial with a JAK inhibitor in PV are now available. Here, we review the current treatment landscape in PV, as well as therapies currently in development.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Aspirin / therapeutic use
  • Forecasting
  • Humans
  • Interferon-alpha / therapeutic use
  • Janus Kinase 2 / antagonists & inhibitors*
  • Janus Kinase 2 / metabolism
  • Phlebotomy / trends
  • Polycythemia Vera / diagnosis*
  • Polycythemia Vera / enzymology
  • Polycythemia Vera / therapy*
  • Treatment Outcome

Substances

  • Interferon-alpha
  • JAK2 protein, human
  • Janus Kinase 2
  • Aspirin