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Aplastic Anaemia

Improved survival in severe acquired aplastic anemia of childhood

Bone Marrow Transplantation volume 26, pages 1149–1156 (2000)Cite this article

Abstract

Multi-agent immunosuppressive therapy has produced improved survival for severe acquired aplastic anemia in children. Recently, some investigators have suggested that immunosuppressive therapy may replace bone marrow transplantation as first-line therapy for this disorder. To assess its validity, we compared the outcomes of bone marrow transplantation vsimmunosuppressive therapy in one institution from 1987 to 1997. We studied 46 consecutive patients less than 18 years of age who presented between January 1987 and April 1997. Inherited marrow failure syndromes and myelodysplastic syndromes were excluded. Patients received immunosuppressive therapy vs bone marrow transplantation based on availability of HLA-matched donors. The main outcome measures were survival, complete marrow and hematological remission, or partial remission but achieving independence from transfusional support. Twenty patients received multi-agent immunosuppressive therapy (cyclosporine, antithymocyte globulin and methylprednisolone); 11 attained complete remission and three partial remission for a transfusion-independent survival of 70%. Six patients died of infectious and hemorrhagic complications. Twenty-six patients were transplanted and 24 (93%) achieved complete remission; one achieved a PR, 25 remain transfusion independent with a median follow-up of 5.9 years or 70 months. One patient developed AML 34 months after successful transplant and one patient died due to graft failure and complications of transplant. There has been a striking improvement in survival for pediatric patients treated with multi-agent immunosuppression in the last decade. However, transplantation results have also improved and this remains the definitive first-line therapy for severe acquired aplastic anemia in this age group. Bone Marrow Transplantation (2000) 26, 1149–1156.

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Author information

Authors and Affiliations

  1. Division of Hematology/Oncology, and the Department of Pediatrics, The Hospital for Sick Children, University of Toronto Faculty of Medicine, Toronto, Ontario, Canada

    M Fouladi, R Herman, M Rolland-Grinton, V Blanchette, S Calderwood, J Doyle, D Halperin, M Leaker, EF Saunders, A Zipursky & MH Freedman

  2. Department of Biostatistics and Epidemiology, St Jude Children's Research Hospital, Memphis, Tennessee, USA

    D Jones-Wallace

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  1. M Fouladi
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  2. R Herman
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Fouladi, M., Herman, R., Rolland-Grinton, M. et al. Improved survival in severe acquired aplastic anemia of childhood. Bone Marrow Transplant 26, 1149–1156 (2000). https://doi.org/10.1038/sj.bmt.1702699

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  • DOI: https://doi.org/10.1038/sj.bmt.1702699

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