Recent Advances and Long-Term Results of Medical Treatment of Acquired Aplastic Anemia: Are Patients Cured?

Horse antithymocyte globulin plus cyclosporine remains standard immunosuppressive therapy in severe aplastic anemia, with hematologic response rates of 60% to 70%. In those refractory to this regimen, a second course of therapy with rabbit antithymocyte globulin plus cyclosporine or alemtuzumab produces responses in 30% to 40%. Eltrombopag, a thrombopoietin receptor agonist, showed activity as a single agent in those refractory to initial immunosuppression, with hematologic response rates of 40% to 50%. When combined with immunosuppression as frontline therapy, eltrombopag increased the rate of overall and complete response rates. Longer follow-up is needed to better define these outcomes.