Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers.

IF 2.7 3区医学 Q3 NEUROSCIENCES

Cerebellum Pub Date : 2024-08-01 Epub Date: 2023-11-13 DOI:10.1007/s12311-023-01625-2

Winfried Ilg, Sarah Milne, Tanja Schmitz-Hübsch, Lisa Alcock, Lukas Beichert, Enrico Bertini, Norlinah Mohamed Ibrahim, Helen Dawes, Christopher M Gomez, Hasmet Hanagasi, Kirsi M Kinnunen, Martina Minnerop, Andrea H Németh, Jane Newman, Yi Shiau Ng, Clara Rentz, Bedia Samanci, Vrutangkumar V Shah, Susanna Summa, Gessica Vasco, James McNames, Fay B Horak

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Abstract

With disease-modifying drugs on the horizon for degenerative ataxias, ecologically valid, finely granulated, digital health measures are highly warranted to augment clinical and patient-reported outcome measures. Gait and balance disturbances most often present as the first signs of degenerative cerebellar ataxia and are the most reported disabling features in disease progression. Thus, digital gait and balance measures constitute promising and relevant performance outcomes for clinical trials.This narrative review with embedded consensus will describe evidence for the sensitivity of digital gait and balance measures for evaluating ataxia severity and progression, propose a consensus protocol for establishing gait and balance metrics in natural history studies and clinical trials, and discuss relevant issues for their use as performance outcomes.

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共济失调试验的定量步态和平衡结果:由共济失调全球倡议工作组关于数字运动生物标志物的共识建议。

随着对退行性共济失调的疾病修饰药物的出现，生态有效，精细颗粒化，数字健康措施非常有必要增加临床和患者报告的结果措施。步态和平衡障碍最常表现为退行性小脑共济失调的第一个迹象，也是疾病进展中报道最多的致残特征。因此，数字步态和平衡测量为临床试验提供了有希望和相关的性能结果。这篇具有嵌入式共识的叙述性综述将描述用于评估共济失调严重程度和进展的数字步态和平衡测量的敏感性的证据，提出在自然史研究和临床试验中建立步态和平衡指标的共识协议，并讨论将其用作表现结果的相关问题。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Cerebellum 医学-神经科学

CiteScore

6.40

自引率

14.30%

发文量

150

审稿时长

4-8 weeks

期刊介绍： Official publication of the Society for Research on the Cerebellum devoted to genetics of cerebellar ataxias, role of cerebellum in motor control and cognitive function, and amid an ageing population, diseases associated with cerebellar dysfunction. The Cerebellum is a central source for the latest developments in fundamental neurosciences including molecular and cellular biology; behavioural neurosciences and neurochemistry; genetics; fundamental and clinical neurophysiology; neurology and neuropathology; cognition and neuroimaging. The Cerebellum benefits neuroscientists in molecular and cellular biology; neurophysiologists; researchers in neurotransmission; neurologists; radiologists; paediatricians; neuropsychologists; students of neurology and psychiatry and others.