Eltrombopag for the treatment of aplastic anaemia What is Eltrombopag? Treatment of aplastic anaemia patients with ATG and Ciclosporin is well-established. Eltrombopag is one of the newer treatments for aplastic anaemia. It works like a normal hormone that the body produces and it is likely that it stimulates the bone marrow stem cell. The complete mechanisms of action in aplastic anaemia are still being determined. Eltrombopag is a tablet taken at home. I have aplastic anaemia - is Eltrombopag for me? Currently Eltrombopag is being used for patients with severe aplastic anaemia as part of the RACE trial for first line treatment. Outside of a clinical trial it is not used as first line treatment (certainly, not in the UK). For other patients it is used when they have not responded to their first line or subsequent treatment. Your doctor will discuss with you and determine your treatment plan that is suitable for you. You can access the leaflet (and print if needed) here for information on any side effects, likely response rates and how Eltrombopag interacts with certain foods. A huge THANK YOU goes to colleagues at the St James' Hospital Haematology Team, Leeds for their enthusiastic help and expertise in developing the leaflet.