Storb R, Santos G W
Clin Haematol. 1983 Oct;12(3):721-37. doi: 10.1016/s0308-2261(83)80007-1.
Marrow transplantation is effective treatment for a number of haematological diseases in patients under the age of 50 who have an HLA-identical sibling donor. It is generally successful when used early in the treatment of aplastic anaemia. It is the only treatment that offers long-term disease-free survival for patients with acute leukaemia who have relapsed at least once, with 10-30 per cent apparent cures. Although still somewhat controversial, it appears also to be the treatment of choice for patients with acute non-lymphoblastic leukaemia in first chemotherapy induced remission and for those with chronic myelogenous leukaemia in the chronic phase since approximately 50-60 per cent of these patients are surviving after marrow transplantation in complete remission, apparently cured. Marrow grafting is the only effective treatment for many patients with inherited immunological-deficiency diseases and certain genetic storage diseases. It is being explored for the therapy of patients with lymphoma, Hodgkin's disease, multiple myeloma, small-cell lung cancer, testicular cancer, ovarian cancer and genetic disorders of haematopoiesis. Cures of congenital Fanconi anaemia, Blackfan-Diamond anaemia, osteopetrosis, and paroxysmal nocturnal haemoglobinuria have been achieved by marrow grafting. Genetic disorders associated with haemolytic anaemia and cyclic neutropenia have been cured by marrow grafting in animals. Target disorders for marrow transplantation in humans are thalassaemia major and sickle cell disease, and, indeed, a first successful transplant for treatment of thalassaemia major has recently been described (Thomas et al, 1982). Marrow transplantation has been limited by the fact that many patients do not have HLA-identical siblings and very few have monozygotic twins. The Seattle team has now explored the use of less well-matched family member donors in more than 80 patients with leukaemia. These donors share one HLA haplotype genetically with the patient and are phenotypically identical at two of the three major HLA loci on the other HLA haplotype (Clift et al, 1979). Overall, the post-transplant survival appears more a reflection of the type and stage of the leukaemia than of the marrow donor. Patients with leukaemia grafted in relapse have a projected survival of 20-30 per cent and those transplanted in remission of 50 per cent. The incidence and severity of GVHD may not be significantly different from that of patients given HLA-identical sibling marrow grafts.(ABSTRACT TRUNCATED AT 400 WORDS)
骨髓移植对于50岁以下且有人类白细胞抗原（HLA）匹配同胞供者的多种血液系统疾病而言是有效的治疗方法。在再生障碍性贫血治疗早期使用时，通常会取得成功。对于至少复发过一次的急性白血病患者，骨髓移植是唯一能提供长期无病生存的治疗方法，约有10% - 30%的患者明显治愈。尽管仍存在一定争议，但对于首次化疗诱导缓解的急性非淋巴细胞白血病患者以及慢性期慢性粒细胞白血病患者，骨髓移植似乎也是首选治疗方法，因为这些患者中约50% - 60%在骨髓移植后处于完全缓解状态，显然已被治愈。骨髓移植是许多遗传性免疫缺陷疾病和某些遗传性贮积病患者的唯一有效治疗方法。目前正在探索将其用于治疗淋巴瘤、霍奇金病、多发性骨髓瘤、小细胞肺癌、睾丸癌、卵巢癌以及造血系统遗传性疾病患者。通过骨髓移植已治愈了先天性范可尼贫血、先天性纯红细胞再生障碍性贫血、骨质石化症和阵发性夜间血红蛋白尿。在动物实验中，与溶血性贫血和周期性中性粒细胞减少相关的遗传性疾病已通过骨髓移植治愈。人类骨髓移植的目标疾病是重型地中海贫血和镰状细胞病，事实上，最近已有首例成功治疗重型地中海贫血的移植报道（托马斯等人，1982年）。骨髓移植受到诸多限制，许多患者没有HLA匹配的同胞，仅有极少数有同卵双胞胎。西雅图研究团队现已对80多名白血病患者使用了匹配度稍差的家庭成员供者进行研究。这些供者与患者在基因上共享一个HLA单倍型，并且在另一个HLA单倍型的三个主要HLA位点中的两个位点上表现型相同（克利夫特等人，1979年）。总体而言，移植后的生存率似乎更多地反映了白血病的类型和分期，而非骨髓供者情况。复发时接受移植的白血病患者预计生存率为20% - 30%，缓解期接受移植的患者为50%。移植物抗宿主病（GVHD）的发生率和严重程度与接受HLA匹配同胞骨髓移植的患者相比可能并无显著差异。（摘要截选至400字）
