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从进展到进步:多发性硬化症的未来。

From progression to progress: The future of multiple sclerosis.

作者信息

Oh Jiwon, Giacomini Paul S, Yong V Wee, Costello Fiona, Blanchette François, Freedman Mark S

机构信息

St. Michael's Hospital, Toronto, ON, Canada.

Montreal Neurological Institute and Hospital, Montreal, QC, Canada.

出版信息

J Cent Nerv Syst Dis. 2024 May 6;16:11795735241249693. doi: 10.1177/11795735241249693. eCollection 2024.

Abstract

Significant advances have been made in the diagnosis and treatment of multiple sclerosis in recent years yet challenges remain. The current classification of MS phenotypes according to disease activity and progression, for example, does not adequately reflect the underlying pathophysiological mechanisms that may be acting in an individual with MS at different time points. Thus, there is a need for clinicians to transition to a management approach based on the underlying pathophysiological mechanisms that drive disability in MS. A Canadian expert panel convened in January 2023 to discuss priorities for clinical discovery and scientific exploration that would help advance the field. Five key areas of focus included: identifying a mechanism-based disease classification system; developing biomarkers (imaging, fluid, digital) to identify pathologic processes; implementing a data-driven approach to integrate genetic/environmental risk factors, clinical findings, imaging and biomarker data, and patient-reported outcomes to better characterize the many factors associated with disability progression; utilizing precision-based treatment strategies to target different disease processes; and potentially preventing disease through Epstein-Barr virus (EBV) vaccination, counselling about environmental risk factors (e.g. obesity, exercise, vitamin D/sun exposure, smoking) and other measures. Many of the tools needed to meet these needs are currently available. Further work is required to validate emerging biomarkers and tailor treatment strategies to the needs of individual patients. The hope is that a more complete view of the individual's pathobiology will enable clinicians to usher in an era of truly personalized medicine, in which more informed treatment decisions throughout the disease course achieve better long-term outcomes.

摘要

近年来,多发性硬化症的诊断和治疗取得了重大进展,但挑战依然存在。例如,目前根据疾病活动和进展对MS表型进行的分类,并未充分反映在MS患者不同时间点可能起作用的潜在病理生理机制。因此,临床医生需要转向基于驱动MS残疾的潜在病理生理机制的管理方法。2023年1月召开了一个加拿大专家小组会议,讨论有助于推动该领域发展的临床发现和科学探索的优先事项。五个关键重点领域包括:确定基于机制的疾病分类系统;开发生物标志物(影像学、体液、数字)以识别病理过程;采用数据驱动的方法整合遗传/环境风险因素、临床发现、影像学和生物标志物数据以及患者报告的结果,以更好地描述与残疾进展相关的诸多因素;利用基于精准的治疗策略针对不同的疾病过程;以及通过接种爱泼斯坦-巴尔病毒(EBV)疫苗、提供有关环境风险因素(如肥胖、运动、维生素D/阳光照射、吸烟)的咨询和其他措施来预防疾病。满足这些需求所需的许多工具目前已经具备。需要进一步开展工作来验证新出现的生物标志物,并根据个体患者的需求调整治疗策略。希望对个体病理生物学有更全面的了解,将使临床医生能够开启一个真正个性化医疗的时代,即在整个疾病过程中做出更明智的治疗决策,从而取得更好的长期效果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9d06/11072059/4e58e2ddf8c5/10.1177_11795735241249693-fig1.jpg

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