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Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):480-488. doi: 10.1182/asheducation-2017.1.480.

What are the current treatment approaches for patients with polycythemia vera and essential thrombocythemia?

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Center of Research and Innovation of Myeloproliferative Neoplasms (CRIMM), Careggi University Hospital/University of Florence, Florence, Italy.


Polycythemia vera (PV) and essential thrombocythemia (ET) are chronic myeloproliferative neoplasms that are characterized by thrombohemorrhagic complications, symptom burden, and impaired survival mainly due to thrombosis, progression to myelofibrosis, and transformation to acute leukemia. In this manuscript, we will review the most recent changes in diagnostic criteria, the improvements in risk stratification, and the "state of the art" in the daily management of these disorders. The role of conventional therapies and novel agents, interferon α and the JAK2 inhibitor ruxolitinib, is critically discussed based on the results of a few basic randomized clinical studies. Several unmet needs remain, above all, the lack of a curative approach that might overcome the still burdensome morbidity and mortality of these hematologic neoplasms, as well as the toxicities associated with therapeutic agents.

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