Advances in the treatment of aplastic anemia

Abstract

Over the last 2 decades, there have been changes in the treatment strategy for patients with acquired severe aplastic anemia (SAA). Immunosuppressive therapy (IST) is still based on the combination of anti-thymocyte globulin (ATG) and cyclosporin, with or without growth factor (G-CSF). Age used to be a significant predictor only in transplantation: it is now also very significant in patients receiving IST. Currently survival after BMT from HLA identical siblings in young patients exceeds 90% after conventional cyclophosphamide conditioning, and unmanipulated bone marrow, which is still preferred over peripheral blood as a stem cell source. Two final important messages: a short interval between diagnosis and treatment will improve results, and patients should be entered on well-designed prospective clinical trials in order to attempt to further improve outcomes.