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Review

Treatment options for essential thrombocythemia and polycythemia vera

, , , &
Pages 41-55 | Published online: 10 Jan 2014
 

Abstract

Polycythemia vera and essential thrombocythemia are the most common chronic myeloproliferative neoplasms; their molecular basis has been appreciated only recently and is briefly discussed in this article. Major causes of morbidity and mortality are represented by arterial and venous thrombosis, as well as by evolution to myelofibrosis or transformation to acute leukemia. Therapy is currently aimed at reducing the rate of thrombosis without increasing the risk of hematologic transformation that might be caused by exposure to cytotoxic drugs. A risk-oriented approach is employed for stratifying patients to the most appropriate therapeutic options. However, results of clinical trials with interferon, and the expected effects of novel drugs selectively targeting the abnormal pathways that are involved in the clonal myeloproliferation, are pushing therapeutic goals from disease control only to cure. These different issues, and current recommendations for treatment, will be discussed in the review.

Financial & competing interests disclosure

This work was supported by Ministero della Università e Ricerca (COFIN 2006067001_003), Istituto Toscano Tumori, and Associazione Italiana per la Ricerca sul Cancro, Milano. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Notes

As published in the Worth Health Organization Classification of Tumors of Haematopoietic and Lymphoid Tissues (4th Edition).

Data from Citation[112].

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