Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor.
Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor.
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通过移植无关捐赠者的骨髓来重建严重的联合免疫缺陷。
DOI：
10.1056/nejm197712152972403
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发表时间：
1977
期刊：
影响因子：
--
通讯作者：
R. Good
中科院分区：
文献类型：
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作者：
R. O'reilly;B. DuPont;S. Pahwa;E. Grimes;E. Smithwick;R. Pahwa;S. Schwartz;J. Hansen;F. Siegal;M. Sorell;A. Svejgaard;C. Jersild;M. Thomsen;P. Platz;P. L'esperance;R. Good
一名患有严重联合免疫缺陷的患者接受了7次来自hla - b兼容和hla - d兼容的非亲属供者的骨髓移植，试图提供免疫重建。前四例移植获得了有限的植入，并显示出初步的免疫功能。第五次移植，低剂量环磷酰胺后，产生细胞介导的免疫重建。骨髓发育不全发生在非致病性菌群再污染后。先前保存在液氮中的骨髓移植是无效的。在高剂量环磷酰胺后进行移植，实现了持久的植入，完全的造血和免疫重建。移植后17个月，功能完整的移植物仍然存在。移植物抗宿主病是慢性和中度严重的，但仅限于皮肤和口腔黏膜。对于缺乏匹配的兄弟姐妹或相关供体的严重联合免疫缺陷或再生障碍性贫血患者，来自无亲缘关系的组织相容性供体的骨髓移植可能是一种有用的治疗方法。
A patient with severe combined immunodeficiency received seven transplants of bone marrow from an HLA-B-compatible and HLA-D-compatible unrelated donor in an attempt to provide immunologic reconstitution. The first four transplants achieved restricted engraftment with evidence of rudimentary immunologic function. A fifth transplant, given after low-dose cyclophosphamide, produced reconstituion of cell-mediated immunity. Marrow aplasia developed after recontamination with a nonpathogenic microflora. Transplantation of marrow previously stored in liquid nitrogen was ineffective. A subsequent transplant, administered after high-dose cyclophosphamide, achieved durable engraftment, with complete hematopoietic and immunologic reconstitution. Seventeen months after transplantation, full functional engraftment persists. Graft-versus-host disease has been chronic and moderately severe, but limited to the skin and oral mucosa. Transplantation of marrow from unrelated histocompatible donors may provide a useful treatment for patients with severe combined immunodeficiency or aplastic anemia who lack a matched sibling or related donor.