Hobbs J R
Westminster Children's Bone Marrow Team, Department of Immunology, Charing Cross and Westminster Medical School, London, United Kingdom.
Eur J Pediatr. 1992;151 Suppl 1:S44-9. doi: 10.1007/BF02125802.
The first paper [9] advocating the displacement use of bone marrow transplantation (DBMT) to treat a variety of genetic metabolic diseases (including thalassaemia major) was put before a European Working Party in 1978. It evolved from mainly Westminster experience which showed the need [6] for DBMT and first successfully used donors other than matched siblings [9]. The principles of using DBMT to install a donor marrow as a component factory which can last a lifetime are outlined. It is not a panacea, being applicable to only about 7% of known inborn errors. Worthwhile correction of some 50 previously disabling diseases in over 700 patients has already been achieved worldwide and for most of the survivors no further treatment is used after 1 year. Guidelines for future extension, including gene transplants, are offered. The superior results of elective DBMT (about 95%) should encourage paediatricians to aim for earlier diagnoses and evaluations for transplants.
1978年，第一篇主张采用置换性骨髓移植（DBMT）治疗多种遗传代谢疾病（包括重型地中海贫血）的论文[9]提交给了一个欧洲工作组。它主要源于威斯敏斯特的经验，该经验表明了DBMT的必要性[6]，并首次成功使用了匹配同胞以外的供体[9]。文中概述了利用DBMT植入供体骨髓作为可终身发挥作用的“组件工厂”的原理。它并非万灵药，仅适用于约7%的已知先天性疾病。全球范围内已经成功矫正了700多名患者中约50种先前导致残疾的疾病，并且对于大多数幸存者来说，1年后无需进一步治疗。文中还提供了包括基因移植在内的未来扩展指南。选择性DBMT的卓越效果（约95%）应促使儿科医生争取更早地进行移植诊断和评估。
