Eltrombopag regimen improves response rates as initial therapy for severe aplastic anemia
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The addition of eltrombopag to standard immunosuppressive therapy appeared safe and increased response rates among transplant-ineligible patients with severe aplastic anemia, according to results of the randomized phase 3 RACE trial.
Researchers presented the findings during the European Society for Blood and Marrow Transplantation’s virtual 46th Annual Meeting.
“Eltrombopag [Promacta, Novartis] is registered in Europe for second-line treatment of aplastic anemia, so it is only available to patients who cannot receive bone marrow transplantation and have failed immunosuppressive treatment,” Régis Peffault de Latour, MD, PhD, professor of hematology in the department of hematology and bone marrow transplant at Saint-Louis Hospital in Paris, said in a press release.
The international, open-label RACE trial aimed to improve standard treatment for patients with severe aplastic anemia.
Investigators randomly assigned 197 patients aged 15 years or older with acquired severe aplastic anemia who had not received prior immunosuppressive therapy to either standard immunosuppression (n = 101; median age, 52 years), which consisted of 40 mg/kg horse antithymocyte globulin (Atgam, Pfizer) four times daily plus 5mg/kg daily cyclosporine A, or standard immunosuppressive therapy plus 150 mg daily eltrombopag (n = 96; median age, 55 years) from day 14 through 6 months or 3 months for patients who had early disease progression.
Patients in the standard treatment-only and eltrombopag combination groups had similar baseline characteristics, including age (younger than 40 years, 35.6% vs. 30.2%), severity of aplastic anemia (very severe, 33.7% vs. 35.4%) and presence of paroxysmal nocturnal hemoglobinuria clone (59.2% vs. 45.2%).
Complete response rates at 3 months served as the primary endpoint.
Median follow-up was 18 months.
Results showed 3-month complete response rates of 21.9% with the eltrombopag combination and 9.9% with standard treatment (pooled OR = 3.2; P = .012), and overall response rates, including complete and partial responses, of 59.4% vs. 31.7%.
Researchers observed a sustained ORR benefit with the eltrombopag combination at 6 months (76.3% vs. 50%; OR = 3.8).
Eltrombopag appeared well-tolerated, with comparable adverse events between the two treatment groups. However, more patients died in the standard treatment group (n = 14 vs. 8).
“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 anemia not eligible for transplantation,” Peffault de Latour and colleagues wrote.
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Peffault de Latour R, et al. Abstract 018. Presented at: European Society for Blood and Marrow Transplantation Annual Meeting (virtual); Aug. 29-Sept. 1, 2020.
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