Recommendations for the diagnosis and treatment of patients with polycythaemia vera

Abstract

Objectives To present the Central European Myeloproliferative Neoplasm Organisation (CEMPO) treatment recommendations for polycythaemia vera (PV). Methods During meetings held from 2015 through 2017, CEMPO discussed PV, and its treatment and recent data. Results PV is associated with increased risks of thrombosis/thrombo‐haemorrhagic complications, fibrotic progression, and leukaemic transformation. Presence of Janus kinase (JAK)‐2 gene mutations is a diagnostic marker and standard diagnostic criterion. World Health Organization 2016 diagnostic criteria for PV, focusing on haemoglobin levels and bone marrow morphology, are mandatory. PV therapy aims at managing long‐term risks of vascular complications and progression towards transformation to acute myeloid leukaemia and myelodysplastic syndrome. Risk stratification for thrombotic complications guides therapeutic decisions. Low‐risk patients are treated first line with low‐dose aspirin and phlebotomy. Cytoreduction is considered for low‐risk (phlebotomy intolerance, severe/progressive symptoms, cardiovascular risk factors) and high‐risk patients. Hydroxyurea is suspected of leukaemogenic potential. IFN‐α has demonstrated efficacy in many clinical trials; its pegylated form is best tolerated, enabling less‐frequent administration than standard interferon. Ropeginterferon alfa ‐2b has been shown to be more efficacious than hydroxyurea. JAK1/JAK2‐inhibitor ruxolitinib is approved for hydroxyurea resistant/intolerant patients. Conclusions Greater understanding of PV is serving as a platform for new therapy development and treatment response predictors. This article is protected by copyright. All rights reserved.