Management of polycythaemia vera: a critical review of current data

• Published in: British journal of haematology Vol. 172; no. 3; pp. 337 - 349
• Main Authors: McMullin, Mary F., Wilkins, Bridget S., Harrison, Claire N.
• Format: Journal Article
• Language: English
• Published: England 01.02.2016
• Subjects: Disease Management; Drug Resistance; Humans; Hydroxyurea - therapeutic use; JAK2; Janus Kinase 2 - antagonists & inhibitors; Janus Kinase 2 - genetics; myeloproliferative; polycythaemia vera; Polycythemia Vera - diagnosis; Polycythemia Vera - drug therapy; Polycythemia Vera - etiology; Polycythemia Vera - genetics; Protein Kinase Inhibitors - therapeutic use; Pyrazoles - therapeutic use; Risk Factors; JAK2; polycythaemia vera; myeloproliferative
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.13812

Summary Polycythaemia vera (PV) is a chronic blood cancer; its clinical features are dominated by myeloproliferation (erythrocytosis, often leucocytosis and/or thrombocytosis) and a tendency for thrombosis and transformation to myelofibrosis or acute myeloid leukaemia. In the past 10 years the pathophysiology of this condition has been defined as JAK/STAT pathway activation, almost always due to mutations in JAK2 exons 12 or 14 (JAK2 V617F). In the same time period our understanding of the optimal management of PV has expanded, most recently culminating in the approval of JAK inhibitors for the treatment of PV patients who are resistant or intolerant to therapy with hydroxycarbamide. It has also been demonstrated that life expectancy for many patients with PV is not normal, nor is their quality of life. We critically explore these findings and discuss their impact. In addition, we highlight persisting gaps in our current management strategy; for example, what is the optimal first line cytoreductive therapy and, indeed, which patients need cytoreductive drugs.