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造血干细胞移植治疗多发性硬化症:最新进展。

Haematopoietic Stem Cell Transplantation for the Treatment of Multiple Sclerosis: Recent Advances.

机构信息

Department of Brain Sciences, Imperial College London, London, UK.

Department of Neurosciences, Drug and Child Health, University of Florence, Florence, Italy.

出版信息

Curr Neurol Neurosci Rep. 2023 Sep;23(9):507-520. doi: 10.1007/s11910-023-01290-2. Epub 2023 Aug 17.

Abstract

PURPOSE OF REVIEW

Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly considered a treatment option for patients with multiple sclerosis (MS), an autoimmune demyelinating and degenerative disease of the central nervous system (CNS). AHSCT persistently suppresses inflammation and improves the disease course in large proportions of patients with relapsing-remitting (RR) MS. Aim of this article is to review the relevant new knowledge published during the last 3 years.

RECENT FINDINGS

Laboratory studies reported confirmatory and new insights into the immunological and biomarker effects of AHSCT. Retrospective clinical studies confirmed excellent outcomes in RRMS, showing possible superior effectiveness over standard therapies and suggesting a possible benefit in early secondary progressive (SP) MS with inflammatory features. New data on risks of infertility and secondary autoimmunity were also reported. Further evidence on the high effectiveness and acceptable safety of AHSCT strengthens its position as a clinical option for aggressive RRMS. Further research is needed to better define its role in treatment-naïve and progressive forms of MS, ideally within randomised clinical trials (RCTs).

摘要

目的综述

自体造血干细胞移植(AHSCT)越来越多地被认为是多发性硬化症(MS)患者的一种治疗选择,MS 是一种中枢神经系统(CNS)的自身免疫性脱髓鞘和退行性疾病。AHSCT 持续抑制炎症并改善大多数复发性缓解型(RR)MS 患者的疾病进程。本文旨在综述过去 3 年发表的相关新知识。

最新发现

实验室研究报告证实了 AHSCT 的免疫学和生物标志物作用的新见解。回顾性临床研究证实了 RRMS 的良好结果,显示出其可能优于标准治疗的疗效,并提示在具有炎症特征的早期继发性进展(SP)MS 中可能有益。还报告了关于生育能力和继发性自身免疫风险的新数据。AHSCT 高效且安全性可接受的进一步证据,使其成为侵袭性 RRMS 的一种临床选择。需要进一步研究以更好地确定其在治疗初发和进展型 MS 中的作用,理想情况下在随机临床试验(RCT)中。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8413/10468923/9d1636beb592/11910_2023_1290_Fig1_HTML.jpg

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