Potential risks after eltrombopag treatment: Thromboembolic complications

Eltrombopag/Eltrombopag is a second-line treatment drug for patients with immune thrombocytopenic purpura (ITP) who face refractory thrombocytopenia. Although eltrombopag is generally considered well tolerated, in rare cases it can cause serious thromboembolic complications.

As an oral thrombopoietin receptor agonist (TPO-RA), eltrombopag is mainly used to treat patients who have failed to respond to traditional treatments such as steroids, immunosuppressants, and splenectomy. However, multiple studies have reported that use of eltrombopag may increase the risk of arterial and venous thrombosis. These complications include deep vein thrombosis, pulmonary embolism, portal vein thrombosis (PVT), extensive cerebral venous sinus thrombosis, ischemic stroke, and myocardial infarction.

A long-term follow-up study showed that thromboembolic events occurred more frequently in patients treated with eltrombopag than in the general population. Over a three-year period, approximately 5% of patients experienced a drug-related thromboembolic event. In addition, the analysis showed that the median time from the first use of TPO-RA to thrombosis was approximately 76.6 days.

Therefore, physicians need to carefully weigh the risks of bleeding versus blood clots in patients receiving eltrombopag. The target platelet count is usually set at 30-50k/μL, which is the optimal balance between bleeding risk and thrombotic risk. A good response to eltrombopag is generally considered when the platelet count reaches 50k/μL, and this goal is usually achieved within two weeks.

In outpatient practice, monitoring of platelet counts is critical to ensure that stable platelet levels are achieved to prevent the formation of blood clots. When the platelet count exceeds150k/μL, the doctor may consider reducing the drug dose, and when the platelet count exceeds 250k/μL, treatment may need to be stopped.