Last year the results of the RACE trial were published, providing brand new insight into how to improve treatment of severe and very severe aplastic anaemia for people who do not have a bone marrow transplant. A new summary of these results has now been published, making them more accessible and easy to understand. 

As many of our community will know, if a suitable donor for bone marrow transplant as initial treatment is unavailable, someone with severe or very severe aplastic anaemia is likely to receive immunosuppression treatment. Standard immunosuppression treatment includes horse antithymocyte globulin (hATG) and ciclosporin A (CsA).

The RACE trial investigated the results of standard immunosuppression treatment (Group A) versus standard immunosuppression treatment with a medication called eltrombopag (Group B) in participants with severe and very severe aplastic anaemia. Eltrombopag is a medicine that improves the blood platelet level and is taken by mouth (orally).

The study found that:

  • Standard immunosuppression treatment (hATG plus CsA) with eltrombopag (Group B) was more beneficial for participants with severe and very severe aplastic anaemia, compared with standard immunosuppression treatment (Group A)
  • The  response  in  participants  with  severe aplastic anaemia after the  addition  of  eltrombopag  to  standard immunosuppression  treatment  was  faster and of higher quality
  • Adding eltrombopag to standard immunosuppression treatment did not cause more side effects
  • Participants with less severe disease and those under 40 years of age showed a higher response to treatment
  • Participants in both groups reported similar overall health and physical, emotional, and social well-being related to their daily activities. 

The findings of this study are very important for patients and can help doctors to consider adding eltrombopag to the standard immunosuppressant treatment (hATG plus ciclosporin for persons who cannot undergo bone marrow transplant as initial treatment.)

A new plain language summary of the RACE trial has been published which is a great resource to help people affected by aplastic anaemia to understand this trial, the results, and what they might mean for people receiving treatment for aplastic anaemia in the future. You can read a summary here, and click through from this page to read the whole PDF:


If you need advice or want to chat after reading about these results, we are here for you. Get in touch with our team