Adding eltrombopag to immunosuppressive therapy improves response in severe aplastic anaemia

January 2022 Pharma News Nalinee Pathak

Researchers have reported higher response rates with the addition of eltrombopag (EPG) to standard immunosuppressive therapy in transplant-ineligible severe aplastic anaemia patients. Results of this phase 3 study were recently published in The New England Journal of Medicine.1

The open-label, international RACE trial enrolled 197 patients with acquired severe aplastic anaemia. These patients were randomly assigned (1:1) in two groups. The participants  in group A (n=101) received standard immunosuppression (40 mg/kg horse anti-thymocyte globulin, four times daily plus 5 mg/kg daily cyclosporine A), or in group B  (n=96) they were administered standard immunosuppressive therapy and EPG (150 mg/daily) for six months (from day 14) or three months for patients with early disease progression. The study‚Äôs primary endpoint was the complete hematologic response (HCR) at three months.

Major findings

The complete response rate at three months was doubled with EPG in group B compared to group A (22% vs 10%, odds ratio [95% CI]: 3.2 [1.3-7.8]; p=0.01). Similarly, a higher overall response rate was observed at six months with EPG combination compared to standard treatment (68% vs 41%). Furthermore, the EPG group had a shorter median time to first response (3 vs 8.8 months) and a higher EFS rate (46% vs 34%). Similar adverse events were observed in both groups. In the standard treatment group one patient and in the EPG combination two patients developed the myelodysplastic syndrome.


The RACE trial demonstrated that combining EPG with standard immunosuppressive treatment improved response rates in previously untreated severe aplastic anaemia patients, with no additional side effects.


  1. Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al. Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022 Jan 6;386(1):11-23. doi: 10.1056/NEJMoa2109965. PMID: 34986284.