New Trends in Nontransplant Therapy for Acquired Aplastic Anemia

Aplastic anemia (AA) is a hematological disease characterized by pancytopenia and hypofunctional bone marrow hematopoiesis. Patients with AA are treated with either immunosuppressive therapy (IST) using anti-thymocyte globulin (ATG) and cyclosporine (CsA) or hematopoietic stem cell transplantation (HSCT), if a matched donor is available. The standard IST regimen for AA patients results in response rates up to 70% and even higher overall survival. However, primary and secondary failures after IST remain frequent, and to date, all attempts aiming to overcome this problem have been unfruitful. The nontransplant therapeutic options for AA have significantly expanded during the last few years. Here, we review the new trends of nontransplant therapy for AA and summarize the current therapeutic effect of AA.