Abstract
Eltrombopag is a small molecule oral thrombopoietin receptor agonist which stimulates the production of blood plateles. It leads to activation of the JAK-STAT signaling pathway and increased proliferation and differentiation of megacaryocytes, and consequently to increase of platelet counts.
Eltrombopag is effective in the treatment of idiopathic thrombocytopenic purpura. It may be used also in the treatment of thrombocytopenia accompanying cirrhosis due to hepatitis C and recently has been shown to stimulate a trilinear hematopoiesis in some patients with aplastic anemia, resulting in increased platelet counts along with red and white blood cell counts.
Eltrombopag treatment is generally well tolerated, with increased liver aminotransferases as the only dose limiting toxicity.