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原发性免疫缺陷病(PID)是一组少见的遗传性疾病,患儿往往发生反复感染,因而生命受到严重威胁。造血干细胞移植是唯一能够治愈该病的方法。目前认为人类白细胞抗原(HLA)相合的同胞是最佳的造血干细胞供者。但随着造血干细胞移植技术的发展,非亲缘HLA相合、单倍体相合供者亦成为PID患者可供选择的造血干细胞来源。由于脐带血干细胞库的建立,脐带血干细胞移植成功治疗PID的例数也日益增加。现就不同供者及移植物来源的造血干细胞在严重联合免疫缺陷病、Wiskott-Aldrich综合征、高IgM综合征以及慢性肉芽肿病的移植治疗的特点及进展进行综述。
Primary immunodeficiency disease (PID) is a rare group of inherited diseases in which children frequently experience repeated infections and their life is seriously threatened. Hematopoietic stem cell transplantation is the only way to cure the disease. Currently considered siblings of human leukocyte antigen (HLA) is the best donor of hematopoietic stem cells. However, with the development of hematopoietic stem cell transplantation technology, non-related HLA haplotype, haploidentical donor has also become a source of hematopoietic stem cells for PID patients. Due to the establishment of umbilical cord blood stem cell bank, umbilical cord blood stem cell transplantation for the successful treatment of PID cases are also increasing. The characteristics and progress of hematopoietic stem cells from different donor and graft sources in the treatment of severe combined immunodeficiency disease, Wiskott-Aldrich syndrome, high IgM syndrome and chronic granulomatous disease are reviewed.