Einstein (São Paulo) (Jun 2011)
Current management of severe acquired aplastic anemia
Abstract
Overall survival in severe aplastic anemia has markedlyimproved in the past four decades due to advances in stem celltransplantation, immunosuppressive therapies and supportive care.Horse anti-thymocyte globulin plus cyclosporine is the standardimmunosuppressive regimen in severe aplastic anemia, and oftenemployed as initial therapy as most are not candidates for a matchedrelated stem cell transplantation. With this regimen, hematologicresponse can be achieved in 60 to 70% of cases, but relapse isobserved in 30 to 40% of responders and clonal evolution in 10 to 15%of patients. Efforts to improve outcomes beyond horse anti-thymocyteglobulin plus cyclosporine have been disappointing, with no significant improvement in the critical parameter of hematologic response, which strongly correlates with long-term survival in severe aplastic anemia. Furthermore, rates of relapse and clonal evolution have also not improved with the development of three drug regimens or with more lymphocytotoxic therapies. Therefore, horse anti-thymocyteglobulin plus cyclosporine remains the standard immunosuppressionof choice as first therapy in severe aplastic anemia. Interestingly,survival has markedly improved over the years in large part due tobetter anti-infective therapy and more successful salvage therapieswith immunosuppression and stem cell transplantation. In this reviewgeneral aspects of diagnosis and management are discussed.
