Skip to main content
Log in

Acquired Aplastic Anaemia

Optimum Immunosuppressive Treatment

  • Practical Therapeutics
  • Published:
Clinical Immunotherapeutics Aims and scope Submit manuscript

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.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Similar content being viewed by others

References

  1. Bacigalupo A. Guidelines for the treatment of severe aplastic anemia. Haematologica 1994; 79: 438–44

    PubMed  CAS  Google Scholar 

  2. Young NS, Alter BP. Definitive treatment of acquired aplastic anemia. In: Young NS, Alter BP, editors. Aplastic anemia acquired and inherited. Philadelphia, PA: WB Saunders Co, 1994: 159–200

    Google Scholar 

  3. Camitta BM, Thomas ED, Nathan DG, et al. Severe aplastic anemia: a prospective study on the effect of early marrow transplantation on acute mortality. Blood 1976; 48: 63–70

    PubMed  CAS  Google Scholar 

  4. Rebellato LM, Gross U, Verbanac K, et al. A comprehensive definition of the major antibody specificities in polyclonal rabbit antithymocyte globulin. Transplantation 1994; 57: 685–94

    Article  PubMed  CAS  Google Scholar 

  5. Marsh JCW, Gordon-Smith EC. The role of antilymphocyte globulin in the treatment of chronic acquired bone marrow failure. Blood Rev 1988; 2: 141–8

    Article  PubMed  CAS  Google Scholar 

  6. Camitta BM, Doney K. Immunosuppressive therapy for aplastic anemia: indications, agents, mechanisms and results. Am J Ped Hematol Oncol 1990; 12: 411–24

    Article  CAS  Google Scholar 

  7. Katsanis E, Ramsay NKC. Treatment of acquired severe aplastic anemia. Am J Ped Hematol Oncol 1989; 11: 360–7

    CAS  Google Scholar 

  8. Bacigalupo A. Treatment of severe aplastic anaemia. Baillieres Clin Haematol 1989; 2: 19–35

    Article  PubMed  CAS  Google Scholar 

  9. Champlin R, Ho W, Gale RP. Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med 1983; 308: 113–8

    Article  PubMed  CAS  Google Scholar 

  10. Camitta BM, O’Reilly RJ, Sensenbrenner L, et al. Antithoracic duct lymphocyte globulin therapy of severe aplastic anemia. Blood 1983; 62: 883–8

    PubMed  CAS  Google Scholar 

  11. Young N, Griffith P, Brittain E, et al. A multicenter trial of antithymocyte globulin in aplastic anemia and related diseases. Blood 1988; 72: 1861–9

    PubMed  CAS  Google Scholar 

  12. Gluckman E, Devergie A, Poros A, et al. Results of immunosuppression in 170 cases of severe aplastic anaemia. Br J Haematol 1982; 51: 541–50

    Article  PubMed  CAS  Google Scholar 

  13. Speck B, Gluckman E, Haak HL, et al. Treatment of aplastic anaemia by antilymphocyte globulin with or without allogeneic bone-marrow infusions. Lancet 1977; 2: 1145–8

    Article  PubMed  CAS  Google Scholar 

  14. Doney KC, Weiden PL, Buckner CD, et al. Treatment of severe aplastic anemia using antilymphocyte globulin with or without infusion of HLA haploidentical marrow. Exp Hematol 1981; 9: 829–34

    PubMed  CAS  Google Scholar 

  15. Marsh JCW, Hows JM, Bryett J, et al. Survival after antilymphocyte globulin therapy for aplastic anemia depends on disease severity. Blood 1987; 70: 1046–52

    PubMed  CAS  Google Scholar 

  16. Bacigalupo A, Hows J, Marsh J, et al. Bone marrow transplantation versus immunosuppression for the treatment of severe aplastic anaemia: a report from the EBMT SAA Working Party. Br J Haematol 1988; 70: 177–82

    Article  PubMed  CAS  Google Scholar 

  17. Schrezenmeier H, Marin P, Raghavachar A, et al. Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol 1993; 85: 371–7

    Article  PubMed  CAS  Google Scholar 

  18. Doney K, Pepe M, Storb R, et al. Immunosuppressive therapy of aplastic anemia: results of a prospective randomized trial of antithymocyte globulin (ATG), methylprednisolone and oxymetholone to ATG, very high-dose methylprednisolone and oxymetholone. Blood 1992; 79: 2566–71

    PubMed  CAS  Google Scholar 

  19. Marsh JCW, Zomas A, Hows JM, et al. Avascular necrosis after treatment of aplastic anaemia with antilymphocyte globulin and high dose methylprednisolone. Br J Haematol 1993; 84: 731–5

    Article  PubMed  CAS  Google Scholar 

  20. Stryckmans PA, Dumont JP, Velu T, et al. Cyclosporine in refractory severe aplastic anemia [letter]. N Engl J Med 1984; 310: 655–6

    Article  PubMed  CAS  Google Scholar 

  21. Wissloff F, Godai HC. Cyclosporine in refractory severe aplastic anemia [letter]. N Engl J Med 1985; 312: 1193

    Google Scholar 

  22. Jacobs P, Wood L, Martell RW. Cyclosporin A in the treatment of severe aplastic anaemia. Br J Haematol 1985; 61: 267–72

    Article  PubMed  CAS  Google Scholar 

  23. Leonard EM, Raefsky E, Griffith P, et al. Cyclosporin therapy of aplastic anaemia, congenital and acquired red cell aplasia. Br J Haematol 1989; 72: 278–84

    Article  PubMed  CAS  Google Scholar 

  24. Hinterberger-Fisher M, Höcker P, Lechner K, et al. Oral cyclosporin A is effective treatment for untreated and also previously immunosuppressed patients with severe bone marrow failure. Eur J Haematol 1989; 43: 136–42

    Article  Google Scholar 

  25. Schrezenmeier H, Schiander M, Raghavachar A. Cyclosporin A in aplastic anemia: report of a workshop. Ann Hematol 1992; 65: 33–6

    Article  PubMed  CAS  Google Scholar 

  26. Gluckman E, Esperou-Bourdeau H, Baruchel A, et al. Multicenter randomized study comparing cyclosporin A alone and antithymocyte globulin with prednisone for treatment of severe aplastic anemia. Blood 1992; 79: 2540–6

    PubMed  CAS  Google Scholar 

  27. Marin P, Nomdedeu B, Rovira M, et al. Cyclosporin A versus antilymphocytic globulin in severe aplastic anaemia. Br J Haematol 1989; 73: 285–6

    Article  PubMed  CAS  Google Scholar 

  28. Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. N Engl J Med 1991; 324: 1297–304

    Article  PubMed  CAS  Google Scholar 

  29. Frickhofen N. Antilymphocyte globulin and methylprednisolone with or without cyclosporine for treatment of aplastic anemia. In: Raghavachar A, Schrezenmeier H, Frickhofen N, editors. Aplastic anemia; current perspectives on pathogenesis and treatment. Wien: Blackwell, 1993: 118–25

    Google Scholar 

  30. Bacigalupo A, Chaple M, Hows J, et al. Treatment of aplastic anaemia with antilymphocyte globulin and methylprednisolone with or without androgens: a randomized trial from the EBMT SAA working party. Br J Haematol 1993; 83: 145–51

    Article  PubMed  CAS  Google Scholar 

  31. Tichelli A, Gratwohl A, Würsch A, et al. Late haematological complications in sever aplastic anaemia. Br J Haematol 1988; 69: 413–8

    Article  PubMed  CAS  Google Scholar 

  32. Socié G, Henry-Amar M, Bacigalupo A, et al. Malignant tumors occuring after treatment of aplastic anemia. N Engl J Med 1993; 329: 1152–7

    Article  PubMed  Google Scholar 

  33. Najean Y, Hagenauer O, for the Cooperative Group for the Study of Aplastic and Refractory Anemias. Long term (5 to 20 years) evolution of non-grafted aplastic anemias. Blood 1990; 76: 2222–8

    PubMed  CAS  Google Scholar 

  34. Young NS, Alter BP. Supportive treatment of aplastic anemia. In: Young NS, Alter BP, editors. Aplastic anemia acquired and inherited. Philadelphia: WB Saunders Co, 1994: 201–15

    Google Scholar 

  35. Najean Y. Long-term follow-up in patients with aplastic anemia. Am J Med 1981; 71: 543–51

    Article  PubMed  CAS  Google Scholar 

  36. Marsh JCW, Socié G, Schrezenmeier H, et al. Haematopoietic growth factors in aplastic anaemia: a cautionary note. Lancet 1994; 344: 172–3

    Article  PubMed  CAS  Google Scholar 

  37. Bacigalupo A, Broccia G, Corda G, et al. Antilymphocyte globulin, cyclosporin, and granulocyte colony-stimulating factor in patients with acquired severe aplastic anemia: a pilot study of the EBMT SAA Working Party. Blood 1995; 85: 1348–53

    PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Rights and permissions

Reprints and permissions

About this article

Cite this article

Socié, G., Bacigalupo, A., Devergie, A. et al. Acquired Aplastic Anaemia. Clin Immunother 4, 9–15 (1995). https://doi.org/10.1007/BF03259068

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/BF03259068

Keywords

Navigation