Pharmacotherapy of essential thrombocythemia

Naseema Gangat, Ayalew Tefferi

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Background: The natural history of essential thrombocythemia is characterized by an increased incidence of thrombotic and hemorrhagic events and, in the long-term, a tendency for disease transformation to myelofibrosis or acute leukemia. Advanced age and a prior history of thrombosis are the major predictors of thrombotic complications. Objective: The aim of this study was to outline the current evidence and the authors' opinion regarding the clinical management of patients with essential thrombocythemia. Methods: The study reviewed the pertinent literature addressing the management options for essential thrombocythemia patients. Results/conclusions: Cytoreductive agents can reduce the rate of thrombotic events, but do not affect the overall survival or rate of disease transformation. Thus, a risk-adapted strategy is recommended for the management of patients with essential thrombocythemia. High-risk patients with essential thrombocythemia should be treated with cytoreductive therapy and low-dose aspirin. Hydroxycarbamide is the agent of choice in most patients: however, interferon-α is a reasonable alternative in young patients, pregnancy or those intolerant of hydroxycarbamide. Low-risk patients can be safely observed. The management of intermediate-risk patients needs to be individualized: however, low-dose aspirin can be used after excluding acquired von Willebrand's disease. In the future we await the role of molecularly targeted therapy with JAK2 inhibitors in high-risk essential thrombocythemia patients.

Original languageEnglish (US)
Pages (from-to)1679-1685
Number of pages7
JournalExpert Opinion on Pharmacotherapy
Issue number10
StatePublished - Jul 2008


  • Aspirin
  • Essential thrombocythemia
  • Hydroxycarbamide
  • Treatment

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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