Eltrombopag improves outcome in patients with severe aplastic anaemia

Eltrombopag improves outcome in patients with severe aplastic anaemia

Eltrombopag appeared to be safe and improve response rates when added to standard immunosuppressive therapy among transplant-ineligible patients with severe aplastic anaemia, according to results from the international, open-label, phase 3 RACE trial.

The findings from the study were presented at the virtual 46th annual meeting of the European Society for Blood and Marrow Transplantation.

The study involved 197 patients aged 15 years or older with acquired severe aplastic anaemia who had not received prior immunosuppressive therapy. The participants were randomly assigned to receive either standard immunosuppression (n = 101), which consisted of 40 mg/kg horse antithymocyte globulin (Atgam) four times daily plus 5mg/kg daily cyclosporine A, or standard immunosuppressive therapy plus 150 mg daily eltrombopag (n = 96) from day 14 through 6 months or 3 months for patients who had early disease progression.

The patients in both the groups had similar baseline characteristics, including age (younger than 40 years, 35.6% vs. 30.2%), the severity of aplastic anaemia (very severe, 33.7% vs. 35.4%) and presence of paroxysmal nocturnal hemoglobinuria clone (59.2% vs. 45.2%).

The findings showed that at 3-month the complete response rate showed 21.9% in cohort with eltrombopag combination and 9.9% in the standard treatment alone group. Researchers noted a sustained overall response rate benefit with the eltrombopag combination at 6 months compared to the standard treatment alone (76.3% vs. 50%; OR = 3.8). Eltrombopag appeared well-tolerated, with comparable adverse events between the two treatment groups.

“Eltrombopag [Promacta] is registered in Europe for second-line treatment of aplastic anaemia, so it is only available to patients who cannot receive bone marrow transplantation and has failed immunosuppressive treatment,” Peffault de Latour, MD, Ph D, professor of haematology in the department of haematology and bone marrow transplant at Saint-Louis Hospital in Paris, said in a press release.

“This practice-changing phase 3 trial supports the combination of eltrombopag plus horse antithymocyte globulin and cyclosporine A as the next first-line standard of care for patients with severe aplastic anaemia not eligible for transplantation,” the researchers wrote.