多发性硬化症的靶向治疗：我们是否知道如何衡量我们是否达到了目标？
Treating to target in multiple sclerosis: Do we know how to measure whether we hit it?
机构信息
Department of Neurology, Medical University of Vienna, Vienna, Austria.
Comprehensive Center for Clinical Neurosciences and Mental Health, Medical University of Vienna, Vienna, Austria.
出版信息
Eur J Neurol. 2024 Dec;31(12):e16526. doi: 10.1111/ene.16526. Epub 2024 Oct 24.
BACKGROUND AND PURPOSE
The rapidly evolving landscape of effective treatment options in multiple sclerosis has led to a shift of treatment objectives towards a treat-to-target approach aiming to suppress disease activity below the level of detectability early during the disease. To enable treat-to-target, a thorough reappraisal of available outcome measures with respect to their ability in this regard is required.
METHODS
To that end, we conducted a comprehensive systematic literature review of more than 1000 studies using PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) 2020 methodology focusing on underlying evidence as well as utility and implementability in clinical practice.
RESULTS
From there, we propose a set of measurable outcomes for everyday routine clinical practice as well as advanced/aspirational measurables requiring additional resources. We also outline remaining knowledge/technology gaps that need to be overcome to enable a treat-to-target approach.
CONCLUSIONS
This work provides the basis for an evidence-based definition of outcome targets for relevant stakeholders and regulatory authorities.
背景与目的
多发性硬化症的有效治疗方案不断发展，治疗目标已转向靶向治疗，旨在在疾病早期将疾病活动抑制到检测不到的水平。为了实现靶向治疗，需要对现有疗效评估方法进行全面重新评估，以评估其在这方面的能力。
方法
为此，我们采用 PRISMA（系统评价和荟萃分析的首选报告项目）2020 方法，对 1000 多项研究进行了全面的系统文献综述，重点关注基础证据以及在临床实践中的实用性和可实施性。
结果
在此基础上，我们提出了一套可用于日常临床实践的可衡量的结果指标，以及需要额外资源的高级/理想的可衡量指标。我们还概述了实现靶向治疗所需克服的遗留知识/技术差距。
结论
这项工作为相关利益相关者和监管机构提供了基于证据的疗效评估目标定义的基础。
Zotero 插件