The addition of cytoreductive therapy does not provide additional benefit to patients with essential thrombocythemia (ET) who are not at high risk for developing thrombosis, according to a recent study published in the Journal of Clinical Oncology.

Previous studies indicated cytoreductive therapy was beneficial for high-risk patents. However, the recent study is one of the first to explore the benefit of cytoreductive therapy in a population of patients with ET not considered to be at high risk of thrombosis.

The study included 382 patients ages 40 to 59 years with no history of ischemia, thrombosis, embolism, hemorrhage, extreme thrombocytosis (platelet count 1500 × 109/L or greater), hypertension, or diabetes requiring therapy. Patients were randomized 1:1 to hydroxycarbamide plus aspirin or aspirin alone.

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At a median follow-up of 73 months and a total follow-up of 2373 patient-years, no significant difference was seen between the 2 groups in terms of overall survival, adverse events, or patient-reported quality of life. Furthermore, no significant differences were seen in the composite end point of transformation to myelofibrosis, acute myeloid leukemia, or myelodysplasia.

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The addition of cytoreductive therapy did not reduce vascular events, myelofibrotic transformation, or leukemic transformation in adult patients with ET who did not have high-risk factors for thrombosis. Therefore, the authors recommend that patients aged 40 to 59 years whose platelet counts are less than 1500 × 109/L and who have no other clinical indications for cytoreductive therapy (such as previous thrombosis or hemorrhage) should not receive cytoreductive therapy.


Godfrey AL, Campbell PJ, MacLean C, et al. Hydroxycarbamide plus aspirin versus aspirin alone in patients with essential thrombocythemia age 40 to 59 years without high-risk features. J Clin Oncol. 2018;36(34):3361-3369.