Department of Neurology, Technische Universität München.
German Center for Neurodegenerative Diseases (DZNE).
Curr Opin Neurol. 2018 Aug;31(4):448-454. doi: 10.1097/WCO.0000000000000581.
This update discusses novel aspects on clinicopathological concepts and therapeutic challenges in progressive supranuclear palsy (PSP) and multiple system atrophy (MSA), arising from publications of the last 1.5 years.
The clinical criteria for diagnosis of PSP have been revised. Clinical variability of pathologically defined PSP and MSA makes the development of mature biomarkers for early diagnosis and biomarker-based trial design indispensable. Novel molecular techniques for biomarker supported diagnosis of PSP and MSA and for monitoring disease progression are being studied. Research in the pathophysiology of both diseases generates gradual progress in the understanding of the underlying processes. Several promising disease-modifying therapeutic approaches for PSP and MSA are now moving into clinical trials.
Recent research generates insights in the pathophysiological relevant processes and raises hope for earlier clinical diagnosis and disease-modifying therapies of patients with PSP and MSA.
本文通过对过去 1.5 年发表的文献进行讨论,阐述了进行性核上性麻痹(PSP)和多系统萎缩(MSA)在临床病理概念和治疗挑战方面的新进展。
PSP 的临床诊断标准已经修订。经病理证实的 PSP 和 MSA 的临床表现存在差异,这使得开发成熟的生物标志物用于早期诊断和基于生物标志物的临床试验设计变得不可或缺。目前正在研究用于 PSP 和 MSA 生物标志物支持诊断和监测疾病进展的新型分子技术。对这两种疾病病理生理学的研究在理解潜在过程方面取得了渐进式的进展。一些有前景的 PSP 和 MSA 疾病修饰治疗方法现已进入临床试验阶段。
最近的研究深入了解了与病理生理相关的过程,并为 PSP 和 MSA 患者的早期临床诊断和疾病修饰治疗带来了希望。