Good R A, Kapoor N, Reisner Y
Cell Immunol. 1983 Nov;82(1):36-54. doi: 10.1016/0008-8749(83)90139-9.
Thus, we can conclude that marrow transplantation has already influenced medical practice greatly. It has offered a treatment which often cures patients of more than 20 otherwise lethal diseases. The treatment so horrendously difficult and dangerous at first has already been greatly improved, simplified, and made much safer. The availability of a suitable donor has been much extended and real progress has been made in prevention and perhaps even in treatment of graft-versus-host disease. This has made possible the option of marrow transplantation for every patient in whom we think the treatment may be beneficial. The problem underlying many cases of interstitial pneumonia has been identified and patients are already benefitting clinically from this progress. Progress has also been made which promises antiviral therapy which could reduce, prevent, and ultimately eliminate the intercurrent virus infections which limit the applicability of marrow transplantation, especially for children with severe immunodeficiencies. I do not know how far this line of investigation can be taken. However, just as we have learned stepwise to use marrow transplants from matched siblings to treat many diseases, to use fetal liver in place of bone marrow, to employ matched relative donors when a matched sibling is not available, and, finally, even to use parental donors to achieve correction of SCID, we now have good reason to believe that, ultimately, we can use marrow transplantation without fear of GVHD to address many additional genetically determined and acquired diseases; certainly, for those diseases that involve any of the cells that are derived from bone marrow cells, and perhaps for those attributable even to cells of other organs and tissues, the functions of which are, in whole or in part, a consequence of interactions of marrow-derived cells and cells of ectodermal or endodermal origin, marrow transplantation may be useful. To us, the future of marrow transplantation as a major modality of treatment or prevention of many diseases, including hemoglobinopathesis, immunodeficiencies, hematologic abnormalities, abnormalities of function of marrow-derived cells, and even inborn errors of function of cells of organs and tissues not of marrow origin, seems bright, indeed. Further, with the capacity to introduce resistance genes against viruses and malignancies, autoimmune diseases, and diseases dependent on anomalies of immune response genes, marrow transplantation for many other diseases seems a more remote possibility.(ABSTRACT TRUNCATED AT 400 WORDS)
因此，我们可以得出结论，骨髓移植已经对医学实践产生了巨大影响。它提供了一种治疗方法，常常能治愈20多种原本致命的疾病。起初极其困难和危险的治疗方法已经有了很大的改进、简化，并且安全性大大提高。合适供体的可及性得到了极大扩展，在移植物抗宿主病的预防甚至治疗方面都取得了实际进展。这使得我们可以为每一位我们认为可能从治疗中受益的患者选择骨髓移植。许多间质性肺炎病例的潜在问题已经被查明，患者已经在临床上从这一进展中受益。在抗病毒治疗方面也取得了进展，有望减少、预防并最终消除限制骨髓移植适用性的并发病毒感染，尤其是对于严重免疫缺陷的儿童。我不知道这一研究方向能走多远。然而，正如我们逐步学会使用匹配同胞的骨髓移植来治疗多种疾病、用胎肝替代骨髓、在没有匹配同胞时使用匹配的亲属供体，甚至最终使用父母供体来纠正重症联合免疫缺陷一样，我们现在有充分的理由相信，最终我们能够使用骨髓移植而不用担心移植物抗宿主病来治疗许多其他遗传性疾病和后天性疾病；当然，对于那些涉及任何源自骨髓细胞的细胞的疾病，甚至对于那些归因于其他器官和组织的细胞的疾病，其功能全部或部分是骨髓衍生细胞与外胚层或内胚层来源细胞相互作用的结果，骨髓移植可能会有用。对我们来说，骨髓移植作为治疗或预防许多疾病的主要方式，包括血红蛋白病、免疫缺陷、血液学异常、骨髓衍生细胞功能异常，甚至非骨髓来源器官和组织细胞的先天性功能缺陷疾病，其前景似乎一片光明。此外，随着引入抗病毒和抗恶性肿瘤、自身免疫性疾病以及依赖免疫反应基因异常的疾病的抗性基因的能力，骨髓移植用于许多其他疾病似乎是一种更遥远的可能性。（摘要截选至400字）
