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Eltrombopag Augments Immunosuppression in Severe Aplastic Anemia

Improvement seen in the rate, rapidity, strength of hematologic response in previously untreated patients with 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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