Letter to the Editor DOI: 10.4274/Tjh-2013.0005 The Impact of Eltrombopag Administration on the Clinical Course of Severe Refractory Fatal Acquired Aplastic Anemia

Severe aplastic anemia (SAA) has an aggressive clinical course and represents a “difficult-to-treat ” situation with current medications [1]. Eltrombopag is a c-mpl receptor agonist oral thrombopoietin-mimetic drug, mainly active in immune thrombocytopenic purpura (ITP) [2]. Single-agent oral eltrombopag produced hematological responses in 11 of 25 cases of aplastic pancytopenia, with trilineage responses observed in some, suggesting a stimulatory effect of early myeloid progenitors in a pilot clinical trial [3]. We would like to share our experience with eltrombopag in 2 patients with SAA refractory to conventional immunosuppressive treatment. Since the thrombopoietin/c-mpl receptor system is present in early hematopoiesis and in hematopoietic stem cells (HSCs) [4], SAA is an area of potential clinical application for thrombopoietin receptor agonists, includin