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Vol. 103, No. 1, 2000
Issue release date: March 2000
Acta Haematol 2000;103:19–25

Current Results of Bone Marrow Transplantation in Patients with Acquired Severe Aplastic Anemia

Report of the European Group for Blood and Marrow Transplantation

Bacigalupo A. · Oneto R. · Bruno B. · Socié G. · Passweg J. · Locasciulli A. · van Lint M.T. · Tichelli A. · McCann S. · Marsh J. · Ljungman P. · Hows J. · Marin P. · Schrezenmeier H.
on behalf of the Working Party on Severe Aplastic Anemia of the European Group for Blood and Marrow Transplantation, and the Medical Statistical Department, University of Leiden, The Netherlands

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We have analyzed 2,002 patients grafted in Europe between 1976 and 1998 from an identical twin (n = 34), from an HLA-identical sibling (n = 1,699) or from an alternative donor (n = 269), which included unrelated and family mismatched donors. The proportions of patients surviving in these three groups are, respectively, 91, 66 and 37%: major causes of failure were acute graft-versus host disease (GvHD) (11%), infection (12%), pneumonitis (4%), rejection (4%). In multivariate Cox analysis, factors predicting outcome were patient’s age (p < 0.0001), donor type (p < 0.0001), interval between diagnosis and bone marrow transplantation (BMT) (p < 0.0005), year of BMT (p = 0.0005) and female donor for a male recipient (p = 0.02). Patients were then divided in two groups according to the year of BMT: up to or after 1990. The overall death rate dropped from 43 to 24% (p < 0.00001). Improvements were seen mostly for grafts from identical siblings (from 54 to 75%, p < 0.0001), and less so for alternative-donor grafts (from 28 to 35%; p = 0.07). Major changes have occurred in the BMT protocol: decreasing use of radiotherapy in the conditioning regimen (from 35 to 24%; p < 0.0001) and increasing use of cyclosporin (with or without methotrexate) for GvHD prophylaxis (from 70 to 98%; p < 0.0001). In conclusion, the outcome of allogeneic BMT for patients with severe aplastic anemia has considerably improved over the past two decades: young patients, grafted early after diagnosis from an identical sibling, have currently an over 80% chance of long-term survival. Transplants from twins are very successful as well. The risk of complications with alternative donor transplants is still high.

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  1. Santos GW, Owens AH Jr: Allogeneic marrow transplants in cyclophosphamide treated mice. Transplant Proc 1969;1:44–46.

    External Resources

  2. Storb R, Thomas ED, Buckner CD, et al: Allogeneic marrow grafting for treatment of aplastic anaemia: A follow-up on long-term survivors. Blood 1976;48:817–841.

    External Resources

  3. Storb R, Prentice RL, Thomas ED: Marrow transplantation for treatment of aplastic anemia. An analysis of factors associated with graft rejection. N Engl J Med 1977;296:61–65.

    External Resources

  4. Storb R, Thomas ED for the Seattle Marrow Transplant Team: Marrow Transplantation for Treatment of Aplastic Anaemia. Clin Haematol 1978;7/3.
  5. Storb R, Erzioni R, Anasetti C, Appelbaum FR, Buckner CD, Besinger W, Bryant E, Clift R, Deeg HJ, Doney K, Flowers M, Hansen J, Martin P, Pepe M, Sale G, Sanders J, Singer J, Sullivan KM, Thomas ED, Witerspoon RP: Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood 1994;84:941–949.

    External Resources

  6. Bacigalupo A, Hows J, Gluckman E, Nissen C, Marsh J, Van Lint MT, Congiu M, de Planque MM, Ernst P, McCann S, Ragavashar A, Frickhofen N, Wursch A, Marmont AM, Gordon-Smith EC, for the EBMT Working Party on Severe Aplastic Anaemia: Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): A report of the EBMT SAA Working Party. Br J Haematol 1988;177:70.
  7. Camitta BM, Thomas ED, Nathan DG, Gale RP, Kopecky KJ, Rapeport JM, Santos G, Gordon-Smith EC, Storb R: A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood 1979;53:504.

    External Resources

  8. Champlin RE, Ho WG, Nimer SD, et al: Bone Marrow transplantation for severe aplastic anemia. Transplantation 1990;49:720–724.

    External Resources

  9. Deeg HJ, Leisenring W, Storb R, Nims J, Flowers ME, Witherspoon RP, Sanders J, Sullivan KM: Long-term outcome after marrow transplantation for severe aplastic anemia. Blood 1998;91:3637–3645.
  10. Doney K, Leisenring W, Storb R, Appelbaum FR: Primary treatment of acquired aplastic anemia: Outcomes with bone marrow transplantation and immunosuppressive therapy. Ann Intern Med 1997;126:107–115.
  11. Gluckman E, Socié G, Devergie A, Bourdeau-Esperou H, Traineau R, Cosset JM: Bone marrow transplantation in 107 patients with severe aplastic anemia using cyclophosphamide and thoraco-abdominal irradiation for conditioning: Long-term follow-up. Blood 1993;78:2451–2455.
  12. McCann SR, Lawler M: Mixed chimaerism: Detection and significance following BMT. Bone Marrow Transplant 1993;11:91–94.
  13. McCann SR, Bacigalupo A, Gluckman E, Hinterberger W, Hows J, Ljungman P, Marin P, Nissen C, van’t Veer Kerthof E, Raghavashar A, Socié G, Frickhofen N, Locasciulli A, Schrezenmeier H: Graft rejection and second bone marrow transplants for acquired aplastic anaemia: A report from the Aplastic Anaemia Working Party of the European Bone Marrow Transplant Group. Bone Marrow Transplant 1994;13:233–237.

    External Resources

  14. McGlave PB, Haake R, Miller W, Kim T, Kersey J, Ramsay NKC: Therapy of severe aplastic anemia in young adults and children with allogeneic bone marrow transplantation. Blood 1987;70:1325–1330.

    External Resources

  15. Passweg JR, Socié G, Hinterberger W, Bacigalupo A, Biggs JC, Camitta BM, Champlin RE, Gale RP, Gluckman E, Gordon-Smith EC, Hows JM, Klein JP, Nugent ML, Pasquini R, Rowlings PA, Speck B, Tichelli A, Zhang MJ, Horowitz MM, Bortin MM: Bone marrow transplantation for severe aplastic anemia: Has outcome improved? Blood 1997;90:858–864.
  16. Speck B, Tichelli A, Gratwohl A, Nissen C: Treatment of severe aplastic anemia: A 12-year follow-up of patients after bone marrow transplantation or after therapy with antilymphocyte globulin; in Shahidi NT (ed): Aplastic Anemia and Other Bone Marrow Failure Syndromes. London, Springer 1990, p 96.
  17. Speck B, Gluckman E, Haak HL, van Rood JJ: Treatment of aplastic anemia by antilymphocyte globulin with and without allogeneic bone marrow infusion. Lancet 1977;ii:1145.
  18. Storb R, Erzioni R, Anasetti C, Appelbaum FR, Buckner CD, Besinger W, Bryant E, Clift R, Deeg HJ, Doney K, Flowers M, Hansen J, Martin P, Pepe M, Sale G, Sanders J, Singer J, Sullivan KM, Thomas ED, Witerspoon RP: Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood 1994;84:941–949.

    External Resources

  19. Socié G, Henry-Amar M, Cosset JM, Devergie A, Girinsky T, Gluckman E: Increased incidence of solid malignant tumors after bone marrow transplantation for severe aplastic anemia. Blood 1991;78:277–279.
  20. Socié G, Amar MH, Bacigalupo A, Hows J, Tichelli A, Ljungman P, McCann S, Frickhofen N, van’t Veer Korthof E, Gluckman E: Malignancies occurring after treatment of aplastic anemia: A survey conducted by the severe aplastic anaemia Working Party of the European Group for Bone Marrow Transplantation. N Engl J Med 1993;329:1152–1157.

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