造血干细胞移植与自身免疫性疾病

Hematopoietic stem cell transplantation for autoimmune disease

目的:认识造血干细胞移植治疗自身免疫性疾病的历史发展及其机制,讨论治疗过程中关于疗效和安全性的相关问题。方法:检索Pubmed数据库和中国期刊全文数据库文献,然后对资料进行初审,并查看每篇文献后的引文。纳入标准:文章所述内容为造血干细胞移植治疗自身免疫性疾病的机制和应用研究;以近5年且发表在较权威杂志者优先。排除标准:重复研究或Meta分析类文章。结果:①造血干细胞移植最早用于治疗血液恶性肿瘤,随着临床医学的发展,其适应证逐步扩展至血液系统疾病、自身免疫性疾病、实体瘤和基因缺陷疾病等。②目前认为造血干细胞移植治疗自身免疫性疾病的作用,主要是建立在大剂量免疫抑制剂对原有免疫系统的清除作用基础上造血干细胞移植后的免疫重建。③造血干细胞移植治疗自身免疫性疾病的疗效与安全性各不相同,与病种、病例的选择、预处理方案有关。结论:①造血干细胞移植治疗自身免疫性疾病尚未确定可获得长期安全稳定临床效应的最佳移植方案。总体认为造血干细胞移植近期疗效显著,而远期疗效尚需多中心、多病例的临床研究和观察,以进一步探讨其安全性和有效性。②只有在真正实现移植后的免疫重建,控制移植后复发,最终才能达到通过造血干细胞移植治愈自身免疫性疾病。

AIM： To analyze the development and mechanism of hematopoietic stem cell transplantation on the treatment of autoimmune disease and to discuss relevant problems of outcome and safety during surgery. METHODS： Pubmed database and China Journal Full-text Database （CJFD） were undertaken. Relevant articles were primarily checked and quotations of each article were searched. Inclusive criteria： articles that were related to application and mechanism of hematopoietic stem cell transplantation for treating autoimmune disease, and were published in recent five years, or in the authorized journals. Exclusive criteria： repetitive studies or Meta analysis articles. RESULTS： ①Hematopoietic stem cell transplantation is performed to treat blood malignant tumor in early phase. With the development of clinical medicine, it can be applied in hematological system disease, autoimmune disease, solid tumor, gene defect disease and so on. ②It is believed that the effect of hematopoietic stem cell transplantation for autoimmune disease mainly depends on immunologic reconstitution after hematopoietic stem cell transplantation on the basis of the clearance of a large dose of immunodepressant in original immune system. ③Outcome and safety of hematopoietic stem cell transplantation for autoimmune disease are various, which is associated with entity, case choice and pretreatment. CONCLUSION： ①The optimal hematopoietic stem cell transplantation method, which can obtain long-term safe stable clinical effect, in the treatment of autoimmune disease is still unclear. Hematopoietic stem cell transplantation has significant short-term outcome. As for long-term effect, we require clinical research and observation with multi-center and multiple cases to further explore the safety and effectiveness. ②Hematopoietic stem cell transplantation can cure autoimmune disease by immunologic reconstitution and controlling recurrence after transplantation.