Addition of Eltrombopag Improves Efficacy of Standard Therapy for Aplastic Anemia

A prospective, investigator-led, multicenter, open-label, randomized Phase III trial compared the efficacy and safety of standard immunosuppressive therapy comprising horse antithymocyte globulin [ATG] plus cyclosporine, with or without addition of Novartis’ oral thrombopoietin receptor agonist Promacta (eltrombopag), as first-line therapy in 197 previously untreated patients with severe aplastic anemia. The primary endpoint was a hematologic complete response at three months.

A complete response at three months was documented in 10 percent of patients in Group A (standard treatment only) and 22 percent of patients in Group B (standard treatment plus eltrombopag); the odds ratio was 3.2 (95 percent confidence interval, 1.3 to 7.8; P = 0.01). At six months, the overall complete plus partial response rate was 41 percent in Group A and 68 percent in Group B. The median times to the first response were 8.8 months (Group A) and 3.0 months (Group B). The incidence of severe adverse events was similar in the two groups.

The investigators concluded that “the 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.”