Godfrey AL, et al. - The therapeutic impacts of hydroxycarbamide plus aspirin vs aspirin alone were assessed in patients aged 40 to 59 years with essential thrombocythemia (ET) and without high-risk factors or extreme thrombocytosis in this open-label, randomized trial. No reduction in vascular events, myelofibrotic transformation, or leukemic transformation was seen as a result of preemptive addition of hydroxycarbamide to aspirin for treating these patients. Based on the findings, researchers recommended against the use of cytoreductive therapy in patients aged 40 to 59 years without other clinical indications for treatment (such as previous thrombosis or hemorrhage) and a platelet count < 1,500 × 10⁹/L.

Methods

• This study included patients aged 40 to 59 years, without a history of ischemia, thrombosis, embolism, hemorrhage, extreme thrombocytosis (platelet count ≥ 1,500 × 10⁹/L), hypertension, or diabetes requiring therapy.
• In all, hydroxycarbamide plus aspirin or aspirin alone was received by 382 patients via random assignment (1:1).
• Time to arterial or venous thrombosis, serious hemorrhage, or death from vascular causes was the composite primary end point.
• Time to first arterial or venous thrombosis, first serious hemorrhage, death, incidence of transformation, and patient-reported quality of life were assessed as secondary end points.

Results

• A median follow-up of 73 months and a total follow-up of 2,373 patient-years was performed.
• The chances of patients reaching the primary end point did not differ significantly between the arms (hazard ratio, 0.98; 95% CI, 0.42 to 2.25; P=1.0).
• A low incidence of significant vascular events was reported, at 0.93 per 100 patient-years (95% CI, 0.61 to 1.41).
• Overall survival did not differ; in the composite end point of transformation to myelofibrosis, acute myeloid leukemia, or myelodysplasia; in adverse events; or in patient-reported quality of life.