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Does Cytoreductive Therapy Improve Outcomes in Lower-Risk Essential Thrombocythemia?

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Cytoreductive therapy may be used to reduce the risk of  hemorrhagic complications, including arterial and venous thrombosis, in patients with essential thrombocythemia. While its benefits have been well established in those with high-risk factors (eg, age ≥ 60 years, history of thrombosis, JAK2 mutation) or extreme thrombocytosis, a recent, randomized study showed no benefit in 382 patients aged 40 to 59 years without high-risk features who were treated with a cytoreductive therapy approach using aspirin alone or in combination with hydroxycarbamide.

After a median follow-up of 73 months and a total follow-up of 2,373 patient-years, the investigators observed no significant difference between the aspirin and aspirin plus hydroxycarbamide arms in the likelihood of patients reaching the composite primary end point of time to arterial or venous thrombosis, serious hemorrhage, or death from vascular causes. There was a low incidence of significant vascular events, at 0.93 per 100 patient-years. There were also no differences between the groups in any of the following: overall survival; composite secondary end point of transformation to myelofibrosis, acute myeloid leukemia, or myelodysplasia; adverse events; or patient-reported quality of life.

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Godfrey AL, Campbell PJ, MacLean C, et al; United Kingdom Medical Research Council Primary Thrombocythemia-1 Study; United Kingdom National Cancer Research Institute Myeloproliferative Neoplasms Subgroup; French Intergroup of Myeloproliferative Neoplasms; and the Australasian Leukaemia and Lymphoma Group. Hydroxycarbamide plus aspirin versus aspirin alone in patients with essential thrombocythemia age 40 to 59 years without high-risk features. J Clin Oncol. 2018 Aug 28:JCO2018788414. doi: 10.1200/JCO.2018.78.8414.

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