Eltrombopag Augments Immunosuppression in Severe Aplastic Anemia

THURSDAY, Jan. 6, 2022 (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, M.D., Ph.D., 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) versus 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 three months was assessed as the primary end point.

The researchers found that 10 and 22 percent 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 six months was 41 and 68 percent 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 two groups had a similar incidence of severe adverse events. A karyotypic abnormality that was classified as myelodysplastic syndrome developed in one and two patients in Groups A and B, respectively; event-free survival was 34 and 46 percent, 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.

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