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肌萎缩侧索硬化症的神经生理学创新:加强诊断、监测和治疗评估

Neurophysiologic Innovations in ALS: Enhancing Diagnosis, Monitoring, and Treatment Evaluation.

作者信息

Donaghy Ryan, Pioro Erik P

机构信息

Department of Neurology, Feinberg School of Medicine, Northwestern University, Chicago, IL 60611, USA.

Djavad Mowafaghian Centre for Brain Health, Division of Neurology, Department of Medicine, University of British Columbia, Vancouver, BC V6T 1Z3, Canada.

出版信息

Brain Sci. 2024 Dec 13;14(12):1251. doi: 10.3390/brainsci14121251.

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive disease of both upper motor neurons (UMNs) and lower motor neurons (LMNs) leading invariably to decline in motor function. The clinical exam is foundational to the diagnosis of the disease, and ordinal severity scales are used to track its progression. However, the lack of objective biomarkers of disease classification and progression delay clinical trial enrollment, muddle inclusion criteria, and limit accurate assessment of drug efficacy. Ultimately, biomarker evidence of therapeutic target engagement will support, and perhaps supplant, more traditional clinical trial outcome measures. Electrophysiology tools including nerve conduction study and electromyography (EMG) have already been established as diagnostic biomarkers of LMN degeneration in ALS. Additional understanding of the motor manifestations of disease is provided by motor unit number estimation, electrical impedance myography, and single-fiber EMG techniques. Dysfunction of UMN and non-motor brain areas is being increasingly assessed with transcranial magnetic stimulation, high-density electroencephalography, and magnetoencephalography; less common autonomic and sensory nervous system dysfunction in ALS can also be characterized. Although most of these techniques are used to explore the underlying disease mechanisms of ALS in research settings, they have the potential on a broader scale to noninvasively identify disease subtypes, predict progression rates, and assess physiologic engagement of experimental therapies.

摘要

肌萎缩侧索硬化症(ALS)是一种上运动神经元(UMNs)和下运动神经元(LMNs)均受累的进行性疾病,最终必然导致运动功能衰退。临床检查是该疾病诊断的基础,序贯严重程度量表用于追踪其进展。然而,缺乏疾病分类和进展的客观生物标志物会延迟临床试验入组、混淆纳入标准并限制对药物疗效的准确评估。最终,治疗靶点参与的生物标志物证据将支持甚至可能取代更传统的临床试验结果指标。包括神经传导研究和肌电图(EMG)在内的电生理工具已被确立为ALS中下运动神经元变性的诊断生物标志物。运动单位数量估计、电阻抗肌电图和单纤维EMG技术为疾病的运动表现提供了更多认识。经颅磁刺激、高密度脑电图和脑磁图越来越多地用于评估上运动神经元和非运动脑区的功能障碍;ALS中较少见的自主神经和感觉神经系统功能障碍也可得到表征。尽管这些技术大多在研究环境中用于探索ALS的潜在疾病机制,但它们有潜力在更广泛范围内无创地识别疾病亚型、预测进展速度并评估实验性治疗的生理参与情况。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/48f8/11674262/d564e8ed163b/brainsci-14-01251-g001.jpg

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