Severe aplastic anemia (SAA) and very severe aplastic anemia (vSAA) are blood diseases of the bone marrow. If a suitable donor for bone marrow transplant as initial treatment is unavailable, standard immunosuppression is used. Standard immunosuppression treatment includes horse antithymocyte globulin (hATG) and cyclosporin A (CsA). This summary investigated the results of standard immunosuppression treatment (Group A) versus standard immunosuppression treatment with a medication called eltrombopag (Group B) in participants with SAA and vSAA. Eltrombopag is a medicine that improves the blood platelet level and is taken by mouth (orally).This Plain Language Summary of Publication article from Immunotherapy summarises a study called RACE looking at a potential treatment for people with severe aplastic anemia and very severe aplastic anemia (blood diseases of the bone marrow). They investigated the effectiveness and safety of adding eltrombopag to the standard treatment for these conditions.

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The original article on which this summary is based is called ‘Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia’, and was published in The New England Journal of Medicine.  

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