This review discusses the therapeutic options for aplastic anemia patients not responding to first-line immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporin, and have severe cytopenias at six months after IST. The authors note that hematopoietic cell transplantation is indicated if refractory aplastic anemia patients are fit and have a suitably matched related or unrelated donor. Patients lacking a fully matched donor should be considered for a second course of ATG plus cyclosporin. Other options the authors recommend include the administration of alemtuzumab or the thrombopoietin mimetic eltrombopag.
Marsh JCW, et al. BloodReview: Options for Patients with Refractory Aplastic Anemia
Dec 2013