Update of hematopoietic cell transplantation for sickle cell disease.
Update of hematopoietic cell transplantation for sickle cell disease.
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DOI：
10.1097/moh.0000000000000136
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发表时间：
2015-05
影响因子：
3.2
通讯作者：
Walters MC
中科院分区：
文献类型：
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作者：
Walters MC
造血细胞移植（HCT）是镰状细胞病（SCD）的治愈性疗法，其很少使用，因为有限的同种异体供体可用性，将治疗扩展到世界上受影响最严重的个体居住的地区所需的有限医疗资源，而SCD专家认为，HCT缺乏证据的严谨性，无论是短时间还是长时间-长期毒性概况，共同可能支持其更广泛的应用。在这次更新中，将介绍最近的进展，重点是供体选择，降低毒性的HCT制剂，以及年轻人的治疗。本文总结了人类白细胞抗原相合同胞供者常规骨髓移植的现状。HCT治疗SCD对几乎所有有人类白细胞抗原匹配同胞供者的儿童都有疗效。这种疗法的未来将取决于扩大可能接受治疗的人数。
Hematopoietic cell transplantation (HCT) is a curative therapy for sickle cell disease (SCD) that is utilized very rarely because of limited allogeneic donor availability, limited healthcare resources needed to expand the treatment to regions in the world where most affected individuals reside, and by a view among SCD experts that HCT lacks the evidential rigor with short and long-term toxicity profiles that together might support its broader application. In this update, recent advances focused on donor selection, reduced toxicity preparation for HCT, and treatment of young adults will be presented. The current status of conventional bone marrow transplantation with a human leukocyte antigen-identical sibling donor is summarized. HCT for SCD is curative in almost all children who have a human leukocyte antigen-matched sibling donor. The future of this therapy will hinge on expanding the number of individuals who might be treated.