Which medications are used in the treatment of polycythemia vera (PV)?

Updated: Sep 20, 2021
  • Author: Srikanth Nagalla, MD, MS, FACP; Chief Editor: Sara J Grethlein, MD, FACP  more...
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One objective of therapy for polycythemia vera (PV) is to control the myeloproliferative activity of this disease. Evidence of an increase in levels of white blood cells and/or platelets and organomegaly indicate uncontrolled myeloproliferative activity that requires a myelosuppressive agent.

Studies by the Polycythemia Vera Study Group (PVSG) have led to the abandonment of long-term therapy with phosphorus-32 (32P) and most alkylating agents (eg, busulfan, chlorambucil), and to the use of hydroxyurea (HU) instead. However, long-term data seem to indicate a possible slight late increase in cases of acute leukemia in patients with PV who are treated with HU for more than 15 years.

Ruxolitinib is a second-line therapy for patients in whom HU is ineffective or poorly tolerated. It is also approved for use in intermediate- or high-risk myelofibrosis, including primary myelofibrosis, post-PV myelofibrosis, and post–essential thrombocythemia myelofibrosis.

Another JAK inhibitor, fedratinib, is indicated for adults with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis.

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