Summary
Aplastic anaemia can be cured either by bone marrow transplantation, which leads to stem cell replacement after a myeloablative conditioning regimen, or with nonreplacement therapy, often referred to as immunosuppressive therapy. Antilymphocyte globulin and cyclosporin are used, either alone or in association, as the cornerstone of immunosuppressive therapy for aplastic anaemia. Both cyclosporin and antilymphocyte globulin used alone induce remission in nearly 50% of patients, and long term follow-up of large cohorts indicates survival rates in the range of 60 to 70%. Immunosuppressive therapy should be first-line treatment for patients without a tissue-identical sibling and for those over 40 to 45 years of age.
However, aplastic anaemia is a rare disease and some questions regarding the optimum immunosuppressive therapy remain to be answered. As a rule in rare diseases, these questions are best answered through multicentre prospective randomised trials, now in progress.
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Socié, G., Bacigalupo, A., Devergie, A. et al. Acquired Aplastic Anaemia. Clin Immunother 4, 9–15 (1995). https://doi.org/10.1007/BF03259068
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DOI: https://doi.org/10.1007/BF03259068