摘要
罕见病虽发病率低，但症状严重，危害性大。多数罕见病无法治疗，极少数患者可通过造血干细胞移植（HSCT）获得根治。脐血于细胞移植（UCBT）有免疫原性低、人类白细胞抗原（HLA）相合程度要求稍低、移植物抗宿主反应发生率低等优点，更适合低体质量儿童。现介绍国内外HSCT，特别是UCBT在原发性免疫缺陷病、遗传代谢病、炎症性肠病和骨髓衰竭综合征等较多发生的罕见病中的应用，希望能有助于提高我国罕见病的治疗水平。
Rare diseases are very rare, but usually have severe symptoms. Some rare diseases are life - threa- tening, Most rare diseases cannot be cured. A very small part of these diseases can be cured by hematopoietic stem cell transplantation （ HSCT）. Umbilical cord blood transplantation（UCBT） is more suitable for children for the weak T cell immunity, the lower request for human leukocyte antigen （HLA） identity type and the lower incidence of graft versus host disease （GVHD）. This article reviewed the published data in the treatment of UCBT in primary immunodeficiency disease, inherited metabolic disease, inflammatory bowel disease and bone marrow failure syndrome, in order to improve the level of rare disease treatment by HSCT,especially for UCBT.
出处
《中华实用儿科临床杂志》
CSCD
北大核心
2016年第15期1135-1139,共5页
Chinese Journal of Applied Clinical Pediatrics
关键词
罕见病
脐血干细胞移植
免疫缺陷病
遗传代谢病
炎症性肠病
骨髓衰竭
Rare diseases
Umbilical cord blood transplantation
Immunodeficiency disease
Inherited metabolic diseases
Inflammatory bowel disease
Bone marrow failure
