SEVERE COMBINED IMMUNODEFICIENCY - TREATMENT BY BONE-MARROW TRANSPLANTATION IN 15 INFANTS USING HLA-HAPLOIDENTICAL DONORS
SEVERE COMBINED IMMUNODEFICIENCY - TREATMENT BY BONE-MARROW TRANSPLANTATION IN 15 INFANTS USING HLA-HAPLOIDENTICAL DONORS
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DOI：
10.1007/bf00451897
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发表时间：
1985-01-01
影响因子：
3.6
通讯作者：
KLEIHAUER, E
中科院分区：
文献类型：
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作者：
FRIEDRICH, W;GOLDMANN, SF;KLEIHAUER, E
在 15 名患有严重联合免疫缺陷 (SCID) 的婴儿中，尝试通过 HLA 单倍体相合父母的骨髓移植 (BMT) 进行免疫重建。为了预防移植物抗宿主病 (GvHD)，使用凝集素凝集和绵羊红细胞形成玫瑰花结来消除骨髓移植物中的污染性 T 淋巴细胞。 13 名患者在未进行细胞减灭预处理的情况下接受了移植；其中 11 种开发出了供体依赖性 T 细胞功能，2 种未能做到这一点。这 2 名和另外 2 名患者中的一名在重复移植前和第一次移植前接受了细胞减灭治疗，在 2 名患者中观察到完全的淋巴造血重建。在接受移植的15名患者中，目前有11人还活着。两名最近接受治疗的患者仍在医院，9 名患者在家中，T 细胞功能稳定。目前已有3例患者体液免疫功能恢复正常。在其他情况下，.gamma。球蛋白定期被替换。 除1例出现短暂性皮肤GvHD外，未观察到急性或慢性GvHD并发症。在大多数 SCID 患者中，在半相合、T 细胞耗尽的 BMT 后，无需 GvHD 即可明显发展 T 细胞功能。特殊的患者需要预处理以允许供体细胞植入，这种方法似乎也有利于体液免疫功能的重建。
In 15 infants with severe combined immunodeficiency (SCID), immunological reconstitution was attempted by bone marrow transplantation (BMT) from HLA-haploidentical parents. To prevent graft vs. host disease (GvHD), marrow grafts were depleted of contaminating T-lymphocytes using lectin agglutination and rosette formation with sheep red blood cells. Thirteen patients received transplants without undergoing prior cytoreductive conditioning; 11 of these developed donor-dependent T-cell functions, 2 failed to do this. One of these 2 and 2 further patients received cytoreductive treatment prior to repeat and to 1st transplants and in 2, complete lymphohemopoietic reconstitution was observed. Of the 15 patients who received transplants, 11 are currently alive. Two recently treated patients remain in the hospital, 9 are at home with stable T-cell functions. Normal humoral immune functions have developed upto now in 3 patients. In the others, .gamma. globulins are regularly substituted. Complications of acute or chronic GvHD were not observed with the exception of 1 case who developed transient GvHD of the skin. In a majority of patients with SCID, T-cell functions can evidently develop without GvHD following haploidentical, T-cell-depleted BMT. Exceptional patients require preconditioning to allow donor cell engraftment, an approach that also appears to facilitate reconstitution of humoral immune functions.