Abstract
A total of 81 severe aplastic anaemia patients, aged 2-63 years, received human leucocyte antigen-matched related marrow grafts after cyclophosphamide + antithymocyte globulin followed by postgrafting methotrexate + cyclosporin. Median follow-up was 9.2 years. Ninety-six per cent of patients had sustained engraftment, 24% developed acute graft-versus-host disease (GVHD), grade II in all but two patients, and 26% developed chronic GVHD; all surviving patients eventually responded to immunosuppressive therapy. Six patients developed cancer: one fatal lymphoma and five carcinomas (all five patients are now free of cancer). Survival was 88%. The regimen appeared well tolerated and effective in heavily pretreated patients with aplastic anaemia.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, P.H.S.
MeSH terms
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Adolescent
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Adult
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Anemia, Aplastic / mortality
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Anemia, Aplastic / therapy*
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Antilymphocyte Serum / therapeutic use*
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Antineoplastic Combined Chemotherapy Protocols / therapeutic use
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Bone Marrow Transplantation*
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Child
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Child, Preschool
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Cyclophosphamide / therapeutic use*
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Cyclosporine / therapeutic use
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Female
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Follow-Up Studies
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Graft vs Host Disease
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Humans
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Immunosuppressive Agents / therapeutic use*
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Karnofsky Performance Status
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Male
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Methotrexate / therapeutic use
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Middle Aged
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Multivariate Analysis
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Survival Rate
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Transplantation Conditioning / methods*
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Transplantation, Homologous
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Treatment Outcome
Substances
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Antilymphocyte Serum
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Immunosuppressive Agents
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Cyclosporine
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Cyclophosphamide
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Methotrexate