多发性硬化症亚群:基于患者报告结果和大量临床样本的数据驱动分组。

IF 4.8 2区 医学 Q1 CLINICAL NEUROLOGY
Alessandro S De Nadai, Ryan J Zamora, Alyse Finch, Deborah M Miller, Daniel Ontaneda, Douglas D Gunzler, Farren S Briggs
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引用次数: 0

摘要

背景:虽然标准临床评估对多发性硬化症患者(PwMS)具有重要价值,但它们在描述患者观点和个体水平症状异质性方面的能力有限:根据患者报告的身体、认知和情绪症状结果(PROs)确定多发性硬化症患者亚组。我们还试图将基于患者报告结果的亚组与人口统计学变量、功能障碍、高血压和吸烟状况、传统的定性多发性硬化症(MS)症状分组以及神经表现测量结果联系起来:方法:我们采用横断面设计,将潜在特征分析(LPA)应用于一个大型PROs数据库;分析样本N = 6619):结果:根据PRO模式,我们确定了九种不同的多发性硬化症亚型。这些亚型主要分为低度、中度和高度行动障碍群组。约 70% 的参与者被归入低度行动障碍组,10% 的参与者被归入中度行动障碍组,20% 的参与者被归入高度行动障碍组。在这些分组中,我们观察到了几种意想不到的模式,例如高行动障碍群组报告了低非行动障碍:本研究强调了推进多发性硬化症精准医疗方法的机会。将PROs与数据驱动方法相结合,可以对症状表现进行成本效益高且个性化的描述,为临床实践和未来的研究设计提供依据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Multiple sclerosis subgroups: Data-driven clusters based on patient-reported outcomes and a large clinical sample.

Background: While standard clinical assessments provide great value for people with multiple sclerosis (PwMS), they are limited in their ability to characterize patient perspectives and individual-level symptom heterogeneity.

Objectives: To identify PwMS subgroups based on patient-reported outcomes (PROs) of physical, cognitive, and emotional symptoms. We also sought to connect PRO-based subgroups with demographic variables, functional impairment, hypertension and smoking status, traditional qualitative multiple sclerosis (MS) symptom groupings, and neuroperformance measurements.

Methods: Using a cross-sectional design, we applied latent profile analysis (LPA) to a large database of PROs; analytic sample N = 6619).

Results: We identified nine distinct MS subtypes based on PRO patterns. The subtypes were primarily categorized into low, moderate, and high mobility impairment clusters. Approximately 70% of participants were classified in a low mobility impairment group, 10% in a moderate mobility impairment group, and 20% in a high mobility impairment group. Within these subgroups, several unexpected patterns were observed, such as high mobility impairment clusters reporting low non-mobility impairment.

Conclusions: The present study highlights an opportunity to advance precision medicine approaches in MS. Combining PROs with data-driven methodology allows for a cost-effective and personalized characterization of symptom presentations. that can inform clinical practice and future research designs.

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来源期刊
Multiple Sclerosis Journal
Multiple Sclerosis Journal 医学-临床神经学
CiteScore
10.90
自引率
6.90%
发文量
186
审稿时长
3-8 weeks
期刊介绍: Multiple Sclerosis Journal is a peer-reviewed international journal that focuses on all aspects of multiple sclerosis, neuromyelitis optica and other related autoimmune diseases of the central nervous system. The journal for your research in the following areas: * __Biologic basis:__ pathology, myelin biology, pathophysiology of the blood/brain barrier, axo-glial pathobiology, remyelination, virology and microbiome, immunology, proteomics * __Epidemology and genetics:__ genetics epigenetics, epidemiology * __Clinical and Neuroimaging:__ clinical neurology, biomarkers, neuroimaging and clinical outcome measures * __Therapeutics and rehabilitation:__ therapeutics, rehabilitation, psychology, neuroplasticity, neuroprotection, and systematic management Print ISSN: 1352-4585
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