Aplastic anaemia: Management

Acquired, idiosyncratic aplastic anaemia (AA) is a rare but potentially fat al haematological disorder. Severe AA constitutes an acute medical emergenc y, and supportive therapy is needed to prevent overwhelming sepsis or a lif e threatening haemorrhage. Specific therapy for the disease includes the ch oice between allogeneic stem cell transplantation (SCT) from an HLA-identic al sibling or immunosuppressive therapy with anti-thymocyte globulin (ATG) and cyclosporin A (CSA). Longterm cure rates of 75-90% are now achieved fol lowing HLA (human leukocyte antigen) identical sibling bone marrow transpla nt. The use of donors other than HLA-id siblings for transplantation in AA remains experimental, Transplantation offers the patient a chance of cure, whilst treatment with immunosuppressive therapy carries a long-term risk of relapse and clonal transformation. The haemopoietic growth factors, apart from granulocyte colony stimulating factor (G-CSF), have been shown to be p otentially toxic when given to patients with AA. A short course of G-CSF ma y be useful to help treat severe infection, but its longer-term use with AT G and CSA remains controversial, Results from immunosuppressive treatment c ontinue to improve with time, as a result of the additional use of CSA with ATG, the use of repeat courses of ATG for non-responders and improvements in the supportive care of patients. (C) 2000 Harcourt Publishers Ltd.