Seminars in Hematology
Volume 37, Issue 1 , Pages 69-80, January 2000

Treatment of acquired severe aplastic anemia: Bone marrow transplantation compared with immunosuppressive therapy-the European group for blood and marrow transplantation experience☆

  • Andrea Bacigalupo

      Affiliations

    • Corresponding Author InformationAddress reprint requests to A. Bacigalupo, MD, Department of Hematology, Ospedak San Martina, Genova, Italy 16132.
    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Ronald Brand

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Rosi Oneto

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Barbara Bruno

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Gerard Sodé

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Jakob Passweg

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Anna Locasciulli

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Maria Teresa Van Lint

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • André Tichelli

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Shaun McCann

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Judith Marsh

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Per Ljungman

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Jill Hows

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Pedro Marin

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands
    • ,
  • Hubert Schrezenmeier

      Affiliations

    • From the Working Party on Severe Aplastic Anemia of the European Blood and Marrow Transplantation Group, Leiden, The Netherlands
    • From the Medical Statistical Department, University of Leiden, Leiden, The Netherlands

Abstract 

Patients with severe aplastic anemia (SAA) can be successfully treated with bone marrow transplantation (BMT) or immunosuppressive therapy (IS). The current outcome using both forms of therapy among 3, 669 patients treated in Europe between 1976 and 1998 is reviewed. Significant progress has been made and the overall risk of failure is now low, with survival rates greater than 80% for both treatments. Chronic graft-versus-host disease (GvHD) remains a problem for BMT patients, and carries a high risk of lethal complications. On the other hand, IS patients are exposed to late failure due to relapse or clonal/malignant diseases. First-line BMT from identical siblings is compared with IS therapy in an intent-to-treat analysis of 1, 765 patients, regardless of subsequent transplant status. The outcome of SAA patients has improved considerably over time and is influenced by patient variables such as severity of the disease and age, but also by the choice of the initial treatment.

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☆ Supported by BIOMED EEC Grant No. 951031 to U.S., an Assodazione Italiana Ricerca contro il Cancro (AIRC) Milano grant to A.B., and by the Assodazione Ricerca Trapianto Midollo Osseo (ARITMO), Geneva.

PII: S0037-1963(00)90031-3

Seminars in Hematology
Volume 37, Issue 1 , Pages 69-80, January 2000

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