(HealthDay News) — Among previously untreated patients with severe aplastic anemia, the addition of eltrombopag to standard immunosuppressive therapy improves the rate, rapidity, and strength of hematologic response, according to a study published in the Jan. 6 issue of the New England Journal of Medicine.

Regis Peffault de Latour, MD, PhD, from the Université de Paris, and colleagues conducted an open-label, randomized, phase 3 trial to compare the efficacy and safety of horse antithymocyte globulin plus cyclosporine alone (Group A; 101 patients) vs with the addition of eltrombopag (Group B; 96 patients) as frontline therapy in patients with severe aplastic anemia who were previously untreated. A hematologic complete response at 3 months was assessed as the primary endpoint.

The researchers found that 10% and 22% of patients had a complete response at three months in Groups A and B, respectively (odds ratio, 3.2). The overall response rate (percentage of patients with a complete or partial response) at 6 months was 41% and 68% in Groups A and B, respectively. The median times to first response were 8.8 and 3.0 months, respectively, in Groups A and B.

The 2 groups had a similar incidence of severe adverse events. A karyotypic abnormality that was classified as myelodysplastic syndrome developed in 1 and 2 patients in Groups A and B, respectively; event-free survival was 34% and 46%, respectively.

“The addition of eltrombopag induced a response that was of higher quality and occurred faster without increasing toxic effects,” the authors write.

The study was partially supported by Novartis, Pfizer, and a grant from Alexion Pharma; Novartis manufactures eltrombopag.

Abstract/Full Text (subscription or payment may be required)

Editorial (subscription or payment may be required)