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非典型帕金森病的干预性试验。

Interventional trials in atypical parkinsonism.

作者信息

Eschlböck S, Krismer F, Wenning G K

机构信息

Department of Neurology, Medical University of Innsbruck, Anichstraße 35, 6020, Innsbruck, Austria.

Department of Neurology, Medical University of Innsbruck, Anichstraße 35, 6020, Innsbruck, Austria.

出版信息

Parkinsonism Relat Disord. 2016 Jan;22 Suppl 1:S82-92. doi: 10.1016/j.parkreldis.2015.09.038. Epub 2015 Sep 25.

Abstract

Atypical parkinson disorders (APD) are rapidly progressive neurodegenerative diseases with a variable clinical presentation that may even mimic Parkinson's disease. Multiple system atrophy (MSA), progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) are commonly summarized under this umbrella term. Significant developments in research have expanded knowledge and have broadened available symptomatic treatments, particularly for the treatment of neurogenic orthostatic hypotension. Nonetheless, symptomatic support still remains limited in all of these disorders. Currently, there exists no effective treatment to delay disease progression and disease-modifying trials have failed to provide coherent and convincing results. Recent trials of rasagiline (in MSA), rifampicin (in MSA), tideglusib (in PSP) and davunetide (in PSP) reported negative results. Nevertheless, large cohorts of patients were recruited for interventional studies in the last few years which improved our understanding of trial methodology in APDs immensely. In addition, remarkable progress in basic research has been reported recently and will provide a solid foundation for future therapeutic trials. In this review, we will summarize published randomized, placebo-controlled clinical trials (RCTs) in APDs. Additionally, the design of ongoing and unpublished interventions will be presented.

摘要

非典型帕金森病(APD)是一类快速进展的神经退行性疾病,临床表现多样,甚至可能酷似帕金森病。多系统萎缩(MSA)、进行性核上性麻痹(PSP)和皮质基底节变性(CBD)通常被归在这个统称之下。研究的重大进展拓展了知识范围,也拓宽了可用的对症治疗方法,尤其是在神经源性体位性低血压的治疗方面。尽管如此,在所有这些疾病中,对症支持仍然有限。目前,尚无有效的治疗方法来延缓疾病进展,疾病修饰试验也未能提供一致且令人信服的结果。最近关于雷沙吉兰(用于MSA)、利福平(用于MSA)、替格列汀(用于PSP)和达武奈肽(用于PSP)的试验均报告了阴性结果。然而,在过去几年中,大量患者被纳入干预性研究,这极大地增进了我们对APD试验方法的理解。此外,最近基础研究也取得了显著进展,将为未来的治疗试验提供坚实基础。在本综述中,我们将总结已发表的关于APD的随机、安慰剂对照临床试验(RCT)。此外,还将介绍正在进行和未发表的干预措施的设计。

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