Polycythemia Vera: Current Management and Emerging Treatment

Abstract

Patients with polycythemia vera (PV) are at risk of thromboembolic complications, progression to secondary myelofibrosis and rarely transformation to secondary acute myeloid leukemia (AML). The current goal of treatment is to reduce the risk of thromboembolic complications including acute vascular events and venous thromboembolism (VTE). Low-dose aspirin, phlebotomy and cytoreduction are the mainstays in the therapeutic algorithms. However, long-term phlebotomy and cytoreduction with hydroxyurea often impair quality of life, leading to non-compliance and treatment intolerance. They do not modify the natural history of PV and secondary myelofibrosis and leukemia transformation remains to be important causes of treatment failure. Discovery of JAK2 V617F mutation in PV has not only revamped its diagnostic algorithm but provided important grounds for the development of JAK inhibitors. Lestaurtinib was the first reported agent being evaluated in phase II clinical trial. Ruxolitinib was initially approved by the US Food and Drug Administration (FDA) for the treatment of myelofibrosis and recently approved for PV based on Phase II/III study showing its clinical efficacies in improving polycythemia, splenomegaly and PV-related symptoms. Pegylated interferon is generally effective, has tolerable toxicity profile and may potentially modify the natural history of PV.