Management of Aplasia

Abstract

Prior to the availability of specific treatment for aplastic anaemia (AA) with antithymocyte globulin (ATG) and cyclosporin or bone marrow transplantation (BMT), most patients died within 6 months of presentation, but the survival of patients with severe AA has improved dramatically over the last two decades and many patients now survive long term. Nevertheless, AA still remains a potentially life threatening disease. Because response to treatment with ATG and cyclosporin is delayed for two to three months and sometimes longer, patients usually need intensive transfusional support and careful attention to infection prophylaxis and treatment, particularly fungal infection. It is important to adopt a careful transfusional policy at diagnosis, in particular to help prevent HLA alloimmunisation which results in refractoriness to platelet transfusions, and sensitisation to minor histocompatibility antigens which is thought to mediate graft rejection between HLA identical siblings. The indications for immunosuppressive therapy or HLA identical sibling BMT are fairly clear, but transplantation from volunteer unrelated donors remains a highly controversial and high risk procedure in patients with AA. The effects of haemopoietic growth factors in AA patients have been perhaps not surprisingly disappointing, and not without significant toxicity, with the exception of granulocyte colony stimulating factor (G-CSF) which is well tolerated clinically. G-CSF may be useful in the treatment of severe resistant infections and it may be of benefit when used after treatment with ATG and cyclosporin.