Recommendations for the diagnosis and treatment of patients with polycythaemia vera

Antónia Hatalova; Jiri Schwarz; Mirjana Gotic; Miroslav Penka; Mikulas Hrubisko; Rajko Kusec; Miklós Egyed; Martin Griesshammer; Maria Podolak-Dawidziak; Andrzej Hellmann; Sergiy Klymenko; Emilia Niculescu-Mizil; Petro E Petrides; Sebastian Grosicki; Matjaz Sever; Nathan Cantoni; Jürgen Thiele; Dominik Wolf; Heinz Gisslinger

doi:10.1111/ejh.13156

Recommendations for the diagnosis and treatment of patients with polycythaemia vera

Eur J Haematol. 2018 Jul 30. doi: 10.1111/ejh.13156. Online ahead of print.

Authors

Antónia Hatalova¹, Jiri Schwarz², Mirjana Gotic³, Miroslav Penka⁴, Mikulas Hrubisko¹, Rajko Kusec⁵, Miklós Egyed⁶, Martin Griesshammer^{7

8}, Maria Podolak-Dawidziak⁹, Andrzej Hellmann¹⁰, Sergiy Klymenko¹¹, Emilia Niculescu-Mizil¹², Petro E Petrides¹³, Sebastian Grosicki¹⁴, Matjaz Sever¹⁵, Nathan Cantoni¹⁶, Jürgen Thiele¹⁷, Dominik Wolf^{18

19}, Heinz Gisslinger²⁰

Affiliations

¹ Clinic of Hematology and Blood Transfusion, University Hospital, Faculty of Medicine, Medical School Comenius University, Slovak Medical University, Bratislava, Slovakia.
² Clinical Section, Institute of Hematology and Blood Transfusion, Institute of Clinical and Experimental Hematology, Charles University, Prague, Czechia.
³ Clinic for Hematology Clinical Center of Serbia, Medical Faculty University of Belgrade, Belgrade, Serbia.
⁴ Department of Clinical Hematology, Masaryk University Hospital, Brno, Czechia.
⁵ Department of Hematology, Dubrava University Hospital, University of Zagreb, Medical School, Zagreb, Croatia.
⁶ Department of Hematology, Somogy County Mór Kaposi General Hospital, Kaposvár, Hungary.
⁷ Department of Hematology, Oncology and Palliative Medicine, Johannes Wesling Academic Medical Center, Minden, Germany.
⁸ University Clinic for Hematology, Oncology and Palliative Medicine, Johannes Wesling Medical Center Minden, University of Bochum, Bochum, Germany.
⁹ Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wróclaw Medical University, Wróclaw, Poland.
¹⁰ Department of Hematology and Transplantology, Medical University Hospital, Gdaňsk, Poland.
¹¹ Department of Medical Genetics, State Institution National Research Center for Radiation Medicine of National Academy of Medical Sciences of Ukraine, Kyiv, Ukraine.
¹² Provita Diagnosis and Treatment Center, Bucharest, Romania.
¹³ Hematology Oncology Center Munich, Ludwig-Maximilian's University, Munich, Germany.
¹⁴ Department of Cancer Prevention, Public School of Health, Silesian Medical University, Katowice, Poland.
¹⁵ Department of Hematology, University Clinical Center, Ljubljana, Slovenia.
¹⁶ Division of Hematology, University Clinic of Medicine, Kantonsspital Aarau, Switzerland.
¹⁷ Institute of Pathology, University of Cologne, Cologne, Germany.
¹⁸ Department of Internal Medicine V, Hematology & Oncology, Innsbruck Medical University, Innsbruck, Austria.
¹⁹ Medical Clinic 3, Oncology, Hematology and Rheumatology, University Hospital of Bonn, Bonn, Germany.
²⁰ Division of Hematology and Blood Coagulation, Department of Internal Medicine I, Medical University of Vienna Hospital, Vienna, Austria.

PMID: 30058088
DOI: 10.1111/ejh.13156

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.

Keywords: cytoreductive therapy; diagnosis; management; myeloproliferative neoplasms; polycythaemia vera; recommendations.

Grants and funding

AOP Orphan Pharmaceuticals AG