Eltrombopag: Role in Cytopenias Following Hematopoietic Stem Cell Transplantation

Eltrombopag is a small molecule oral agonist of the thrombopoietin receptor. Initially used for improving thrombocytopenia in chronic immune thrombocytopenia (ITP), it was later found to be efficacious in various other etiologies of thrombocytopenia as well as inherited marrow failure syndromes. Lately, it has been used for thrombocytopenia and poor graft function after allogeneic hematopoietic stem cell transplantation (HSCT) without any severe adverse events. Although prospective evidence of the efficacy is limited, there are increasing reports on the safety and efficacy with Eltrombopag in post HSCT thrombocytopenia and poor graft function. This provides an exciting opportunity for further research to evaluate both efficacy and cost-effectiveness of the use of Eltrombopag in this scenario. Here we review the current evidence on the indications for the use of Eltrombopag in the post allogeneic hematopoietic stem cell transplant setting.