Eltrombopag, a thrombopoietin mimetic, has recently emerged as an encouraging and promising agent for patients with refractory aplastic anemia of various causes (AA). It has demonstrated efficacy in restoring trilineage hematopoiesis, and this positive effect continues after discontinuation of the drug. While there are still ongoing trials, for example, Eltrombopag, a thrombopoietin mimetic, has recently emerged as an encouraging and promising agent for patients with refractory AA. It has demonstrated efficacy in restoring trilineage hematopoiesis, and this positive effect continues after discontinuation of the drug. While there are still ongoing trials, for example, Eltrombopag for Moderate Aplastic Anemia, there is also some literature to support its use. Killick published guidelines in 2015 and lists this drug for APlastic Anemia: “Eltrombopag is licensed by the EMA for SAA refractory to IST or patients who are heavily pre‐treated and unsuitable for HSCT.”.
Lum SH, Grainger JD. Eltrombopag for the treatment of aplastic anemia: current perspectives. Drug Des Devel Ther. 2016;10:2833–2843. Published 2016 Sep 13.
AlessandroBusca et al, Eltrombopag for the Treatment of Refractory Pure RBC Aplasia after Major ABO Incompatible Hematopoietic Stem Cell Transplantation
Biology of Blood and Marrow Transplantation Volume 24, Issue 8, August 2018, Pages 1765-1770
S.B, Killick et al, Guidelines for the diagnosis and management of adult aplastic anaemia. BJH Volume172, Issue2
January 2016 Pages 187-207