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Prognosis and treatment of essential thrombocythemia

Author
Ayalew Tefferi, MD
Section Editor
Stanley L Schrier, MD
Deputy Editor
Alan G Rosmarin, MD

INTRODUCTION

Essential thrombocythemia (ET) is a BCR-ABL1-negative myeloproliferative neoplasm (MPN) characterized by excessive, clonal platelet production. While at least half of patients with ET are asymptomatic at the time of diagnosis, the majority will develop vasomotor, thrombotic, or hemorrhagic manifestations at some point during the course of their disease.

The prognosis and management of ET will be reviewed here. An overview of the MPNs, as well as discussions of the pathologic mechanisms, clinical manifestations, and diagnosis of ET, are presented separately. (See "Overview of the myeloproliferative neoplasms" and "Diagnosis and clinical manifestations of essential thrombocythemia".)

The general approach to the patient with an elevated platelet count is also discussed separately. (See "Approach to the patient with thrombocytosis".)

GOALS OF CARE

The goal of treatment of ET is to prevent thrombotic and hemorrhagic complications and to alleviate symptoms (eg, headaches, dizziness, visual disturbances, burning dysesthesia). Available treatment options are not curative and have not been shown to prolong survival nor prevent disease transformation to acute myeloid leukemia (AML) or post-ET myelofibrosis. (See "Diagnosis and clinical manifestations of essential thrombocythemia", section on 'Clinical features'.)

PRETREATMENT EVALUATION

All patients with ET should have a complete history and physical examination that documents symptoms, signs, and laboratory studies that may alter management strategy. It is our practice to include an evaluation of the following:

                                        

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Literature review current through: Nov 2016. | This topic last updated: Tue Nov 29 00:00:00 GMT 2016.
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