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Outcome of Displacement Bone Marrow Transplantation for Inborn Errors of Metabolism
Authors:JR Hobbs MD
Institution:Westminster Bone Marrow Team, Westminster Hospital, London
Abstract:The concept of displacement bone marrow transplantation arose from our work at Westminster 1970–1973, by which time we had extended the donors from matched siblings to other family and unrelated donors. DBMT is not a panacea, but can be applied to about 7% of understood inborn errors, where it is possible to devise in vitro tests to predict in vivo donor effects. Its use to install donor bone marrow as a component factory for the life of the recipient, and the importance of immunoprophylaxis are summarised. Worthwhile correction has been achieved for 48 previously fatal genetic diseases, partial correction for another five, but there has been failure for three diseases. Some 80% of our patients were not found in known families and could not have been prevented.
Keywords:Displacement bone marrow transplantation  Immunoprophylaxis  Half-matched donors  Unrelated donors              In vivo enzyme transfer  Immunodeficiencies  Mucopolysaccharidoses  Lysosomal disorders  DNA repai defects
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