Eltrombopag Thrombopoietin Receptor Agonist for Treatment of Immune Thrombocytopenia: Current and Future Considerations

Summary: Chronic immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet count that has persisted for more than 12 months. Patients may be asymptomatic but those with severe, disease may have significant morbidity and require treatment. Historically, the pathogenesis of ITP was believed to be increased platelet destruction by anti-platelet antibodies. Treatment options were therefore primarily aimed at reducing platelet autoantibody production or inhibiting macrophage-mediated platelet destruction. These treatments usually have only a transient effect and are often associated with serious treatment related adverse events. Recently, impaired platelet production and inappropriately low serum thrombopoietin levels were observed in many chronic ITP patients. Therefore, stimulation of platelet production by growth factor or growth factor analogues may be useful in ITP treatment. This article presents data on the pharmacology, clinical efficacy, safety profile of Eltrombopag, a novel orally active, thrombopoietin-receptor agonist, in the treatment of ITP.