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未来 MS 队列特征:苏格兰多中心首发队列研究多发性硬化症复发缓解型。

FutureMS cohort profile: a Scottish multicentre inception cohort study of relapsing-remitting multiple sclerosis.

机构信息

Anne Rowling Regenerative Neurology Clinic, The University of Edinburgh Centre for Clinical Brain Sciences, Edinburgh, UK

Chromatin Lab, Genome Regulation Section, The University of Edinburgh MRC Human Genetics Unit, Edinburgh, UK.

出版信息

BMJ Open. 2022 Jun 29;12(6):e058506. doi: 10.1136/bmjopen-2021-058506.

Abstract

PURPOSE

Multiple sclerosis (MS) is an immune-mediated, neuroinflammatory disease of the central nervous system and in industrialised countries is the most common cause of progressive neurological disability in working age persons. While treatable, there is substantial interindividual heterogeneity in disease activity and response to treatment. Currently, the ability to predict at diagnosis who will have a benign, intermediate or aggressive disease course is very limited. There is, therefore, a need for integrated predictive tools to inform individualised treatment decision making.

PARTICIPANTS

Established with the aim of addressing this need for individualised predictive tools, FutureMS is a nationally representative, prospective observational cohort study of 440 adults with a new diagnosis of relapsing-remitting MS living in Scotland at the time of diagnosis between May 2016 and March 2019.

FINDINGS TO DATE

The study aims to explore the pathobiology and determinants of disease heterogeneity in MS and combines detailed clinical phenotyping with imaging, genetic and biomarker metrics of disease activity and progression. Recruitment, baseline assessment and follow-up at year 1 is complete. Here, we describe the cohort design and present a profile of the participants at baseline and 1 year of follow-up.

FUTURE PLANS

A third follow-up wave for the cohort has recently begun at 5 years after first visit and a further wave of follow-up is funded for year 10. Longer-term follow-up is anticipated thereafter.

摘要

目的

多发性硬化症(MS)是一种中枢神经系统免疫介导的神经炎症性疾病,在工业化国家,它是导致工作年龄段人群进行性神经功能障碍的最常见原因。虽然该病可治疗,但疾病活动和对治疗的反应存在很大的个体间差异。目前,在诊断时预测哪些患者将具有良性、中等或侵袭性疾病过程的能力非常有限。因此,需要综合预测工具来为个体化治疗决策提供信息。

参与者

为了满足对个体化预测工具的需求,成立了 FutureMS 研究。这是一项全国代表性、前瞻性观察队列研究,纳入了 440 名在苏格兰居住的新诊断为复发缓解型多发性硬化症的成年人,他们在 2016 年 5 月至 2019 年 3 月期间诊断时处于疾病缓解期。

迄今为止的研究结果

该研究旨在探索 MS 疾病异质性的发病机制和决定因素,并将详细的临床表型与影像学、遗传和生物标志物的疾病活动和进展指标相结合。目前已完成招募、基线评估和 1 年随访。在这里,我们描述了队列设计,并介绍了基线和 1 年随访时参与者的概况。

未来计划

该队列的第 3 次随访波最近在首次就诊后 5 年开始,另外一次随访波的资金已到位,用于第 10 年的随访。此后预计将进行更长期的随访。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1065/9244691/53c56dbd1813/bmjopen-2021-058506f01.jpg

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