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5例多发性硬化症患者造血干细胞移植后的9年随访

Nine-year follow up after hematopoietic stem cell transplantation in five multiple sclerosis patients.

作者信息

Comini-Frota Elizabeth R, Marques Bruna C C, Torres Caio, Cohen Karoline M S, Miranda Eduardo Carvalho

机构信息

Universidade José do Rosário Vellano, Belo Horizonte, MG, Brasil.

Ecoar Centro de Imagens, Belo Horizonte MG, Brasil.

出版信息

Arq Neuropsiquiatr. 2019 Sep 5;77(8):531-535. doi: 10.1590/0004-282X20190097.

DOI:10.1590/0004-282X20190097
PMID:31508677
Abstract

Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system. Its treatment has focused on inflammation control as early as possible to avoid disability. Autologous hematopoietic stem cell transplantation (AHSCT) has been used for treating MS since 1996, with recent decisive results regarding benefits in long-term efficacy. Five patients followed up at an MS center in Belo Horizonte, Brazil, who had relapsing-remitting MS with high disease activity, underwent AHSCT between 2009 and 2011. They were evaluated clinically, with magnetic resonance imaging, and by the EDSS every six months after transplantation, up to July 2018. The patients were four women and one man, with ages ranging from 25-50 years, and time since disease onset ranging from 4-17 years at the time of the procedure. Four patients improved, one patient was stabilized, and all patients were free of disease activity after 5-9 years. Through improving patient selection and decreasing the time from disease onset, AHSCT could stop epitope spreading and disease progression. Despite multiple other therapeutic choices being approved for relapsing-remitting MS, AHSCT continues to be a treatment to consider for aggressive MS disease.

摘要

多发性硬化症(MS)是一种中枢神经系统的免疫介导性疾病。其治疗重点在于尽早控制炎症以避免残疾。自1996年以来,自体造血干细胞移植(AHSCT)一直用于治疗MS,近期在长期疗效方面取得了决定性成果。在巴西贝洛奥里藏特的一家MS中心对5例患者进行了随访,这些患者患有复发缓解型MS且疾病活动度高,于2009年至2011年间接受了AHSCT。在移植后每6个月对他们进行临床评估、磁共振成像检查以及扩展残疾状态量表(EDSS)评估,直至2018年7月。患者中有4名女性和1名男性,年龄在25至50岁之间,手术时疾病发病时间为4至17年。4例患者病情改善,1例患者病情稳定,所有患者在5至9年后均无疾病活动。通过改进患者选择并缩短疾病发病时间,AHSCT可以阻止表位扩展和疾病进展。尽管复发缓解型MS已获批多种其他治疗选择,但AHSCT仍是侵袭性MS疾病值得考虑的一种治疗方法。

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Nine-year follow up after hematopoietic stem cell transplantation in five multiple sclerosis patients.5例多发性硬化症患者造血干细胞移植后的9年随访
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引用本文的文献

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Stem Cell Transplantation for Multiple Sclerosis: A 2023 Review of Published Studies.干细胞移植治疗多发性硬化症:2023年已发表研究综述
Cureus. 2023 Oct 30;15(10):e47972. doi: 10.7759/cureus.47972. eCollection 2023 Oct.
2
The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis.自体造血干细胞移植治疗多发性硬化症的现状。
J Neurol. 2022 Jul;269(7):3937-3958. doi: 10.1007/s00415-022-11063-5. Epub 2022 Apr 11.
3
Effect of autologous hematopoietic stem cell transplantation on multiple sclerosis and neuromyelitis optica spectrum disorder: a PRISMA-compliant meta-analysis.
自体造血干细胞移植对多发性硬化和视神经脊髓炎谱系疾病的影响:一项符合 PRISMA 原则的荟萃分析。
Bone Marrow Transplant. 2020 Oct;55(10):1928-1934. doi: 10.1038/s41409-020-0810-z. Epub 2020 Feb 4.