Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency
Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency
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DOI：
10.1056/nejm199902183400703
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发表时间：
1999-02-18
影响因子：
158.5
通讯作者：
Ward, FE
中科院分区：
文献类型：
--
作者：
Buckley, RH;Schiff, SE;Ward, FE
背景自1968年以来，人们就知道骨髓移植可以改善严重的联合免疫缺陷，但有关该治疗长期疗效的数据有限。我们对1982年5月至1998年9月在杜克大学医学中心接受造血干细胞移植的89例重症联合免疫缺陷婴儿的免疫功能进行了前瞻性研究。骨髓耗尽的T细胞凝集与大豆凝集素和绵羊红细胞复位transplantation.Results前77名婴儿接受T细胞耗尽，HLA半相合的父母marrow，和12个收到HLA相合的骨髓从相关的捐助者; 3的收件人的半相合的骨髓也收到胎盘血移植无关的捐助者。除了两名接受胎盘血的患者外，没有一名接受者在移植前接受化疗或预防移植物抗宿主病。在89名婴儿中，72名（81%）在移植后3个月至16.5年仍然存活，包括所有12名接受HLA相同骨髓的婴儿，77名接受单倍体骨髓的婴儿中的60名（78%），以及3名接受单倍体骨髓和胎盘血的婴儿中的2名（67%）。在接受未分级HLA相同骨髓的患者中，T细胞功能在移植后两周内恢复正常，但在接受T细胞耗尽骨髓的患者中，通常要到移植后三到四个月才恢复正常。在最近一次评估时，72名幸存者中除4名外，其余所有人的T细胞功能正常，他们血液中的所有T细胞都来自供体。B细胞功能在许多单倍体相合骨髓的接受者中仍然异常。在26名儿童中（5名接受HLA相同的骨髓，21名接受单倍体相同的骨髓），2%至100%的B细胞来自供体。45的72名儿童接受静脉注射免疫球蛋白。结论骨髓移植相关捐助者是一种挽救生命和维持生命的治疗与任何类型的严重联合免疫缺陷患者，即使没有HLA相同的捐助者。（N Engl J Med 1999;340：508-16.）（C）1999年。马萨诸塞州医学会。
Background Since 1968 it has been known that bone marrow transplantation can ameliorate severe combined immunodeficiency, bur data on the longterm efficacy of this treatment are limited. We prospectively studied immunologic function in 89 consecutive infants with severe combined immunodeficiency who received hematopoietic stem-cell transplants at Duke University Medical Center between May 1982 and September 1998.Methods Serum immunoglobulin levels and lymphocyte phenotypes and function were assessed and genetic analyses performed according to standard methods. Bone marrow was depleted of T cells by agglutination with soybean lectin and by sheep-erythrocyte resetting before transplantation.Results Seventy-seven of the infants received T-cell-depleted, HLA-haploidentical parental marrow, and 12 received HLA-identical marrow from a related donor; 3 of the recipients of haploidentical marrow also received placental-blood transplants from unrelated donors. Except for two patients who received placental blood, none of the recipients received chemotherapy before transplantation or prophylaxis against graft-versus-host: disease. Of the 89 infants, 72 (81 percent) were still alive 3 months to 16.5 years after transplantation, including all of the 12 who received HLA-identical marrow, 60 of the 77 (78 percent) who were given haploidentical marrow, and 2 of the 3 (67 percent) who received both haploidentical marrow and placental blood. T-cell function became normal within two weeks after transplantation in the patients who received unfractionated HLA-identical marrow but usually not until three to four months after transplantation in those who received T-cell-depleted marrow. At the time of the most recent evaluation, all but 4 of the 72 survivors had normal T-cell function, and all the T cells in their blood were of donor origin. B-cell function remained abnormal in many of the recipients of haploidentical marrow. In 26 children (5 recipients of HLA-identical marrow and 21 recipients of haploidentical marrow) between 2 percent and 100 percent of B cells were of donor origin. Forty-five of the 72 children were receiving intravenous immune globulin.Conclusions Transplantation of marrow from a related donor is a life-saving and life-sustaining treatment for patients with any type of severe combined immunodeficiency, even when there is no HLA-identical donor. (N Engl J Med 1999;340:508-16.) (C)1999. Massachusetts Medical Society.