Experience with bone marrow transplantation for inborn errors of metabolism.
Westminster experience had by 1973 evolved the concept of displacement bone marrow transplantation and extended the donors from matched siblings to other family and unrelated donors. The principles of its use to install donor bone marrow as a component factory for the life of the recipient, together with the importance of immunoprophylaxis are detailed. Satisfying correction has been achieved for 48 previously fatal genetic diseases, partial correction for another 5 with failure for 3 diseases. Displacement bone marrow transplantation is not a panacea, but could be applied to about 7% of known inborn errors, devising in vitro tests which can predict in vivo donor effects, especially since some 80% of our patients are not found in known families and could not have been prevented.
Hobbs
J R
JR
Westminster Hospital, London, UK.
D016026:Bone Marrow Transplantation; D006801:Humans; D008661:Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy*