Eltrombopag in long-term management of pediatric thrombocytopenia.

Eltrombopag is a thrombopoietin receptor agonist that may be effective in a broad range of thrombocytopenias of distinct etiology. We have observed rapid, robust, and sustained responses to eltrombopag in two young patients with refractory thrombocytopenia, one with primary immune thrombocytopenia and the other with Evans syndrome.