Uses

Eltrombopag is a thrombopoietin receptor agonist indicated for thrombocytopenia in adults and pediatric patients (≥1 year) with chronic immune thrombocytopenia (ITP) unresponsive to other therapies, thrombocytopenia in chronic hepatitis C patients to enable antiviral therapy initiation, and severe aplastic anemia.

Do not use if

No absolute contraindications are listed in the FDA label. Use with caution in patients with hepatic impairment, chronic hepatitis C, or those at elevated risk for thromboembolic events.

Side effects

Common adverse effects include nausea, vomiting, myalgia, headache, and upper respiratory tract infections. Hepatotoxicity (elevated liver enzymes) is an important risk. Thromboembolic events, cataracts, and bone marrow reticulin deposition with long-term use have been reported.

Warnings

Carries a boxed warning for hepatic decompensation risk in chronic hepatitis C patients. Monitor liver function tests every two weeks during titration. Avoid in patients with myelodysplastic syndromes due to risk of progression to acute myeloid leukemia.

How it works

Eltrombopag interacts with the transmembrane domain of the human thrombopoietin receptor (c-Mpl), triggering signaling cascades that induce megakaryocyte proliferation and differentiation, thereby increasing platelet production. It does not compete with thrombopoietin for receptor binding.

Storage

Store at room temperature, 20°C to 25°C (68°F to 77°F). Keep out of reach of children.