Pharmacotherapy of polycythemia vera

Introduction: Polycythemia vera (PV) is a clonal myeloproliferative neoplasm associated with arterial and/or venous thromboses and long-term risks of leukemic or myelofibrotic transformation. Areas covered: The authors discuss the state-of-the-art treatment for PV, including the choice of hematocrit target, the use of aspirin and prescription of hydroxyurea, interferon and other cytoreductive agents. This review highlights current areas of need and discusses how pegylated forms of interferon and Janus kinase inhibitors may fit into the treatment armamentarium. Expert opinion: Recent advances in treatment have reduced the risk of major thromboses or cardiovascular death to ∼ 1% per year. In contrast, the risk of leukemic or myelofibrotic transformation remains ever present, and this risk becomes increasingly relevant as thrombosis-related morbidity and mortality decline. Future therapies will need to demonstrate an impact on malignant clone size, and/or on the risk of disease transformation, if further survival prolongations are to be achieved.