Nouh Claire Delpirou, Younes Kyan
Department of Neurology, Division of Behavioral Neurology, Stanford Neuroscience Health Center, 453 Quarry Road, Palo Alto, CA 94304, USA.
Curr Treat Options Neurol. 2024 Jul;26(7):319-338. doi: 10.1007/s11940-024-00797-4. Epub 2024 Jun 25.
The purpose of this review is to discuss the clinical, radiological, and neuropathological heterogeneity of corticobasal syndrome (CBS), which can complicate the determination of underlying etiology and lead to inaccurate treatment decisions. Though the most common diagnosis is corticobasal degeneration (CBD), the spectrum of underlying pathologies expands beyond CBD and can overlap with other neurodegenerative diseases and even the neuroimmunology field. We will review possible clinical presentations and cues that can point towards the etiology. We will also discuss the most recent available biomarkers to facilitate a more accurate diagnosis. Additionally, we will examine current and future potential therapeutic options.
The range of available fluid and neuroimaging biomarkers is increasing and some are already being used in clinical practice. While the treatment of neurodegenerative diseases is largely aimed at managing symptoms, early detection and accurate diagnosis are crucial for initiating early management and enrollment in clinical trials. The recent approval of a disease-modifying therapy for Alzheimer's disease (AD) has raised hopes for the development of more therapeutic options for other proteinopathies. Several candidates are currently being studied in clinical trial pipelines, particularly those targeting tau pathology.
Recent advancements in understanding the genetic and neuropathological diversity of CBS, along with the promising development of fluid and imaging biomarkers, are driving clinical trial research forward, instilling optimism for creating more effective disease-modifying treatments for brain proteinopathies.
本综述旨在探讨皮质基底节综合征(CBS)的临床、影像学和神经病理学异质性,这些异质性可能使潜在病因的确定复杂化,并导致不准确的治疗决策。尽管最常见的诊断是皮质基底节变性(CBD),但其潜在病理范围超出了CBD,可能与其他神经退行性疾病甚至神经免疫学领域存在重叠。我们将回顾可能指向病因的临床表现和线索。我们还将讨论最新可用的生物标志物,以促进更准确的诊断。此外,我们将研究当前和未来潜在的治疗选择。
可用的体液和神经影像学生物标志物的范围正在扩大,其中一些已在临床实践中使用。虽然神经退行性疾病的治疗主要旨在控制症状,但早期检测和准确诊断对于启动早期管理和参与临床试验至关重要。最近一种治疗阿尔茨海默病(AD)的疾病修饰疗法获得批准,为开发针对其他蛋白质病的更多治疗选择带来了希望。目前有几种候选药物正在临床试验阶段进行研究,特别是那些针对tau病理的药物。
最近在理解CBS的遗传和神经病理学多样性方面取得的进展,以及体液和影像学生物标志物的良好发展,正在推动临床试验研究向前发展,为开发更有效的针对脑蛋白质病的疾病修饰疗法注入了乐观情绪。