Hobbs J R
Westminster Hospital, London, UK.
Enzyme. 1987;38(1-4):194-206. doi: 10.1159/000469205.
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.
到1973年，威斯敏斯特医院的经验发展出了替代骨髓移植的概念，并将供体范围从匹配的同胞扩展到其他家庭成员和无关供体。文中详细阐述了将供体骨髓作为受体生命组成部分进行植入的使用原则，以及免疫预防的重要性。对于48种先前致命的遗传疾病已实现了满意的矫正，另有5种疾病得到部分矫正，3种疾病矫正失败。替代骨髓移植并非万灵药，但可应用于约7%的已知先天性疾病，设计体外测试以预测体内供体效应，特别是因为我们约80%的患者并非来自已知家族，无法预防。
