Marrow transplantation from human leukocyte antigen-identical or haploidentical donors for correction of Wiskott-Aldrich syndrome.
Marrow transplantation from human leukocyte antigen-identical or haploidentical donors for correction of Wiskott-Aldrich syndrome.
复制标题
来自人类白细胞抗原相同或半相合供体的骨髓移植用于纠正 Wiskott-Aldrich 综合征。
DOI：
10.1016/s0022-3476(05)83041-0
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发表时间：
1991
期刊：
影响因子：
--
通讯作者：
O'Reilly,RJ
中科院分区：
文献类型：
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作者：
Brochstein,JA;Gillio,AP;Ruggiero,M;Kernan,NA;Emanuel,D;Laver,J;Small,T;O'Reilly,RJ
自1979年以来，共有17例Wiskott-Aldrich综合征患者在纪念斯隆-凯特琳癌症中心接受了异基因骨髓移植。11例患者接受了人类白细胞抗原（HLA）基因型相同的兄弟姐妹（9例）或HLA表型相同的父母（2例）的骨髓。6例患者接受了来自HLA不同父母的骨髓移植。对HLA相合的受者用白消安和环磷酰胺进行细胞减灭术，对不相合的受者进行全身照射，然后用大剂量阿糖胞苷治疗。所有11例HLA相合骨髓受体均成功移植，11例中有10例在移植后28至145个月存活。1例患者在移植后10个月死于慢性移植物抗宿主病和巨细胞病毒引起的间质性肺炎。6例不匹配的移植受者中只有1例存活，移植后52个月以上;其他患者死于广泛的慢性移植物抗宿主病（1例）、淋巴瘤（3例）或进行性全血细胞减少伴无菌血症（1例）。因此，骨髓移植代表了具有HLA相同供体的Wiskott-Aldrich综合征患者的治疗选择。然而，对于缺乏组织相容性家族供体的患者，我们的方法需要修改，以克服同种异体耐药，并减少这些患者的移植后免疫功能不全。
Since 1979, a total of 17 patients with Wiskott-Aldrich syndrome have undergone allogeneic bone marrow transplantation at Memorial Sloan-Kettering Cancer Center. Eleven patients received marrow from either human leukocyte antigen (HLA) genotypically identical siblings (nine patients) or an HLA phenotypically identical parent (two patients). Six patients received marrow grafts from HLA-disparate parents. Cytoreduction was accomplished with busulfan and cyclophosphamide for the HLA-identical recipients and total-body irradiation followed by high-dose cytarabine therapy in the mismatched recipients. All 11 recipients of HLA-identical marrow had successful grafts, and 10 of 11 are alive and well 28 to 145 months after transplantation. One patient died 10 months after transplantation of chronic graft-versus-host disease and interstitial pneumonitis caused by cytomegalovirus. Only one of the six mismatched graft recipients survives, 52+ months after transplantation; the other patients have died of extensive chronic graft-versus-host disease (one patient), lymphoma (three patients), or progressive pancytopenia accompanyingCandidasepsis (one patient). thus bone marrow transplantation represents the treatment of choice in patients with Wiskott-Aldrich syndrome who have an HLA-identical donor. However, our approach for patients lacking a histocompatible family donor requires modifications to overcome allogeneic resistance and decrease the posttransplantation immunoincompetence in these patients.