Dickinson A M, Reid M M, Abinun M, Peak J, Brigham K, Dunn J, Cant A J
Department of Haematology, Royal Victoria Infirmary, Newcastle upon Tyne, UK.
Bone Marrow Transplant. 1997 Feb;19(4):323-9. doi: 10.1038/sj.bmt.1700669.
Bone marrow transplantation is the only curative treatment for children with severe combined immunodeficiency (SCID). In the absence of an HLA-identical sibling, haploidentical parental donor marrow can be used provided it is depleted of T cells to prevent otherwise inevitable GVHD. Campath 1M has been successfully used for this procedure in several centres. In our centre 17 SCID patients plus one with combined immunodeficiency (CID) were transplanted with Campath 1M T cell-depleted bone marrow. Progenitor cell recovery, before and after T cell depletion, was monitored using granulocyte-macrophage colony-forming cell assays (GMCFU) and CD34 analysis. The numbers of GMCFU/kg transplanted correlated with engraftment and survival post-transplant and monitoring CD34+ cell numbers in the T cell-depleted marrow pretransplant may be an additional indicator of successful engraftment. Use of a buffy coat marrow preparation with restriction of the number of T cells to < 5 x 10(5)/kg was associated with graft failure in four and death in five of eight children, probably because too few stem cells were infused. T cell depletion of a mononuclear cell preparation of donor marrow with no arbitrary ceiling of infused T cells is highly effective at preventing clinically important GVHD and cured nine out of 10 children transplanted with such material.
骨髓移植是重症联合免疫缺陷(SCID)患儿唯一的治愈性治疗方法。在没有 HLA 匹配同胞供者的情况下,可以使用单倍体相合的亲代供者骨髓,前提是对其进行 T 细胞清除以预防不可避免的移植物抗宿主病(GVHD)。Campath 1M 已在多个中心成功用于此程序。在我们中心,17 例 SCID 患者加 1 例联合免疫缺陷(CID)患者接受了 Campath 1M 清除 T 细胞的骨髓移植。使用粒细胞-巨噬细胞集落形成细胞测定(GMCFU)和 CD34 分析监测 T 细胞清除前后祖细胞的恢复情况。每千克移植的 GMCFU 数量与移植后的植入和生存相关,移植前监测清除 T 细胞的骨髓中 CD34+细胞数量可能是植入成功的另一个指标。使用血沉棕黄层骨髓制剂,将 T 细胞数量限制在<5×10(5)/kg,8 名儿童中有 4 名出现移植失败,5 名死亡,可能是因为注入的干细胞太少。对供者骨髓的单个核细胞制剂进行 T 细胞清除,且对注入的 T 细胞数量没有任意上限,在预防具有临床重要性的 GVHD 方面非常有效,10 名接受此类材料移植的儿童中有 9 名治愈。