Bone Marrow Failure Disorders

In this prospective, investigator-led, open-label, multicenter, randomized phase 3 trial (ClinicalTrials.gov Identifier: NCT02099747) published in the New England Journal of Medicine, collaborators from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation compared the efficacy and safety of immunosuppressive treatment (horse antithymocyte globulin [ATG] plus cyclosporine) with or without eltrombopag as front-line therapy in previously untreated patients with severe aplastic anemia. The primary end point was a hematologic complete response at 3 months. Median time to first response was 8.8 months with immunosuppressive therapy alone (group A) vs 3 months with immunosuppressive therapy plus eltrombopag (group B). At 3 months, 10% of patients in Group A and 22% in Group B had a complete response (odds ratio, 3.2; 95% CI, 1.3-7.8; P =.01). At 6 months, the overall response rate was 41% in Group A and 68% in Group B. The incidence of adverse events was similar in the 2 groups, and event-free survival was 34% and 46%, respectively. The investigators concluded that "addition of eltrombopag to standard immunosuppressive therapy improved the rate, rapidity, and strength of hematologic response among previously untreated patients with severe aplastic anemia, without additional toxic effects."