Parr M D, Messino M J, McIntyre W
Department of Pharmacy, University Hospital of Arkansas, Little Rock 72205.
Am J Hosp Pharm. 1991 Jan;48(1):127-37.
Bone marrow transplantation (BMT) is discussed in terms of immunology, procedures, and complications and their treatment. Any patient with a disorder of the hematopoietic or immune system or a disease in which a transferable hematopoietic cell can supply a missing enzyme is a candidate for BMT. A priority in allogeneic BMT is the identification of a compatible donor through matching of human lymphocyte antigens (HLAs). The greater the disparity in HLAs, the greater the chance of rejection. The ideal donor is a monozygotic twin or an HLA-matched sibling, but only 30% of patients have such a donor. Before receiving the bone marrow infusion, patients must be conditioned to create space in the marrow for donor cells, suppress the immune system, and eradicate any tumor in patients with malignancies. Conditioning is achieved by the combination of total body irradiation and cyclophosphamide treatment; busulfan, etoposide, and cytarabine have also been used. For patients given unmanipulated marrow, the number of nucleated cells infused is about 3 X 10(8) per kilogram. Signs of engraftment are usually seen 14-21 days later. Toxic effects related to conditioning appear during this period and include infection, gastroenteritis, mucositis, and congestive heart failure. The most serious complication is graft-versus-host disease (GVHD), which can affect multiple organ systems. Prednisone, methylprednisolone, methotrexate, antithymocyte globulin, and cyclosporine have been used in an effort to prevent or treat GVHD. Bone marrow transplantation offers the chance of long-term survival to many patients with terminal disease, but associated morbidity and mortality rates remain high. Research is needed to address the problems of infection, leukemic relapse, and GVHD and the difficulty in obtaining and matching donors.
本文从免疫学、操作程序、并发症及其治疗方面对骨髓移植进行了讨论。任何患有造血或免疫系统疾病，或患有可通过可转移造血细胞提供缺失酶的疾病的患者，都是骨髓移植的候选对象。异基因骨髓移植的一个首要任务是通过人类淋巴细胞抗原（HLA）配型来确定合适的供体。HLA差异越大，排斥的可能性就越大。理想的供体是同卵双胞胎或HLA配型相符的兄弟姐妹，但只有30%的患者有这样的供体。在接受骨髓输注之前，患者必须进行预处理，以便为供体细胞在骨髓中腾出空间、抑制免疫系统，并根除恶性肿瘤患者体内的任何肿瘤。预处理通过全身照射和环磷酰胺治疗相结合来实现；白消安、依托泊苷和阿糖胞苷也已被使用。对于接受未处理骨髓的患者，每公斤输注的有核细胞数量约为3×10⁸ 。通常在14 - 21天后出现植入迹象。在此期间会出现与预处理相关的毒性作用，包括感染、肠胃炎、粘膜炎和充血性心力衰竭。最严重的并发症是移植物抗宿主病（GVHD），它可影响多个器官系统。已使用泼尼松、甲泼尼龙、甲氨蝶呤、抗胸腺细胞球蛋白和环孢素，以预防或治疗GVHD。骨髓移植为许多终末期疾病患者提供了长期生存的机会，但相关的发病率和死亡率仍然很高。需要开展研究来解决感染、白血病复发、GVHD以及获取和配型供体困难等问题。
