title: Eltrombopag in aplastic anemia.
abstract: The treatment of aplastic anemia is currently with immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) and cyclosporine, to which two thirds of patients respond. However, a significant proportion of these responders relapse and many have persistent cytopenias. The management of these patients is challenging. Modifications to this standard approach using alternative immunosuppressive agents or adding hematopoietic cytokines such as granulocyte colony-stimulating factor (G-CSF) and erythropoietin (EPO) have not improved outcome. A recent trial has shown that eltrombopag, a thrombopoeitin mimetic, is efficacious in the treatment of patients with severe aplastic anemia (SAA) refractory to IST. There is evidence that this drug works by directly stimulating marrow stem and progenitor cells thereby promoting hematopoietic recovery in patients with bone marrow failure. Several trials are ongoing in our institution using this very promising drug in combination therapy in the upfront treatment of SAA, in IST-refractory SAA and in moderate disease.
authors.affiliation: Department of Haematology, Tallaght Hospital, Dublin, Ireland; National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Bethesda, MD. Electronic address: ronan.desmond@amnch.ie.
authors.forename: Ronan
authors.initials: R
authors.lastname: Desmond
authors.affiliation: National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Bethesda, MD.
authors.forename: Danielle M
authors.initials: DM
authors.lastname: Townsley
authors.affiliation: National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Bethesda, MD.
authors.forename: Cynthia
authors.initials: C
authors.lastname: Dunbar
authors.affiliation: National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Bethesda, MD.
authors.forename: Neal S
authors.initials: NS
authors.lastname: Young
mesh_terms: D000741:Anemia, Aplastic / Q000188:drug therapy*; D000818:Animals; D001565:Benzoates / Q000627:therapeutic use*; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D011720:Pyrazoles / Q000627:therapeutic use*; D012008:Recurrence; D012307:Risk Factors; D013926:Thrombopoietin / Q000494:pharmacology
chemical_list: D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D013926:Thrombopoietin; C520809:eltrombopag