Introduction: Eltrombopag is an orally bioavailable, non-peptide thrombopoietin receptor agonist capable of stimulating platelet production through the differentiation of CD34+ hematopoietic progenitor cells into committed CD41+ megakaryocyte precursors and proliferation of megakaryocyte progenitor cells.
Areas covered: This drug has been tested in several clinical trials in adult patients with chronic immune thrombocytopenia (ITP), demonstrating the ability of the drug to reduce the burden of thrombocytopenia and its associated side effects. Two multicenter trials on eltrombopag in chronic ITP of childhood have been recently completed, showing that the drug is effective also in pediatric patients. Recent studies have suggested a potential role of eltrombopag in the treatment of thrombocytopenia associated with hepatitis-C virus infection. These studies have documented that adjunct treatment with eltrombopag can help avoid either dose reductions or withdrawal of pegylated interferon due to development of thrombocytopenia. Eltrombopag has shown efficacy also in patients with acquired severe aplastic anemia and myelodysplastic syndromes.
Expert opinion: Eltrombopag plays an important therapeutic role in many different conditions characterized by persistent thrombocytopenia. A more comprehensive definition of both long-term safety and benefits deriving from the use of eltrombopag will be obtained through prolonged observation of patients already enrolled in the different studies conducted so far and from future prospective controlled trials.
Keywords: bleeding disorders; eltrombopag; immune thrombocytopenia; severe aplastic anemia; thrombocytopenia.