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Polycythaemia Vera and Essential Thrombocythaemia

Current Treatment Strategies

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Abstract

Polycythaemia vera (PV) and essential thrombocythaemia (ET) are classified as Philadelphia-negative chronic myeloproliferative diseases. Both PV and ET are rare diseases, but the prevalence is high. Patients who have not been treated for the diseases are at great risk of morbidity and mortality as a result of thrombohaemorrhagic events. However, if patients have been well treated, their prognosis is good and life-expectancy approaches normal.

This article provides diagnostic tools and flowcharts for treatment of PV and ET. Treatment of PV and ET should be risk-adjusted and individualised. Low-dose aspirin is recommended as an antiaggregative drug in both diseases. For PV, phlebotomy to control a haematocrit at <0.45 is the cornerstone in treatment, and treatment with hydroxycarbamide (hydroxyurea) or interferon (IFN)-α is added to reduce hypermetabolic symptoms or splenomegaly becoming cytoreductive. In ET, hydroxycarbamide and anagrelide are the most used drugs, and anagrelide may also be added in PV to reduce thrombocytosis. IFNα is the only myelosuppressive treatment available during pregnancy. Current controversies regarding treatment illustrate the need for more randomised clinical trials. Demonstration of over expression of the PV-1 gene and in particular the JAK-2 mutation will be novel diagnostic criteria and may have an impact for future therapy of both PV and ET.

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Acknowledgements

Dr Penninga has no conflicts of interest that are directly relevant to the content of this review. Dr Bjerrum has participated in scientific meetings and conferences sponsored financially by producers of interferon and anagrelide. No sources of funding were used to assist in the preparation of this review.

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  1. Department of Haematology, National University Hospital, Rigshospitalet, Blegdamsvej 9, 2100, Copenhagen, Denmark

    Elisabeth I. Penninga & Ole W. Bjerrum

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Correspondence to Elisabeth I. Penninga.

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Penninga, E.I., Bjerrum, O.W. Polycythaemia Vera and Essential Thrombocythaemia. Drugs 66, 2173–2187 (2006). https://doi.org/10.2165/00003495-200666170-00003

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