干细胞移植在自身免疫性疾病中的应用。
Application of stem cell transplantation in autoimmune diseases.
机构信息
Department of Rheumatology and Immunology, Singapore General Hospital, Singapore.
Department of Medicine, Duke University Medical Center, Durham, North Carolina, USA.
出版信息
Curr Opin Hematol. 2019 Nov;26(6):392-398. doi: 10.1097/MOH.0000000000000531.
Abstract
PURPOSE OF REVIEW
Autologous hematopoietic stem cell transplantation (HSCT) is a promising therapeutic modality for severe autoimmune diseases. In this review, we will outline the immunological mechanisms and the clinical evidence and experiences for therapeutic HSCT in autoimmune diseases, with particular focus on systemic sclerosis and multiple sclerosis.
RECENT FINDINGS
Approximately 3000 patients with autoimmune diseases worldwide have been treated with HSCT. HSCT in systemic sclerosis has been shown in three randomized controlled trials to be associated with significant long-term event-free survival despite some transplant-related mortality in the first year. A recent controlled trial in multiple sclerosis has also show benefit with transplant.
SUMMARY
The aim of HSCT is to 'reset' one's immune system into a naïve and self-tolerant state through immune depletion and regulation. HSCT requires careful patient selection, close collaboration between physicians and expertise of transplant team to ensure optimal outcome.
摘要
目的综述
自体造血干细胞移植（HSCT）是治疗严重自身免疫性疾病的一种很有前途的治疗方法。在这篇综述中，我们将概述自身免疫性疾病治疗性 HSCT 的免疫学机制和临床证据及经验，特别关注系统性硬化症和多发性硬化症。
最近的发现
全球约有 3000 名自身免疫性疾病患者接受了 HSCT 治疗。三项随机对照试验表明，系统性硬化症 HSCT 与显著的长期无事件生存率相关，尽管在第一年有一些与移植相关的死亡率。最近一项多发性硬化症的对照试验也显示出移植的益处。
总结
HSCT 的目的是通过免疫耗竭和调节“重置”一个人的免疫系统，使其进入幼稚和自身耐受状态。HSCT 需要仔细的患者选择、医生之间的密切合作和移植团队的专业知识，以确保最佳结果。
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