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
{"title":"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。","authors":"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","doi":"10.1007/s12311-023-01625-2","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":50706,"journal":{"name":"Cerebellum","volume":null,"pages":null},"PeriodicalIF":2.7000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11269489/pdf/","citationCount":"0","resultStr":"{\"title\":\"Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers.\",\"authors\":\"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\",\"doi\":\"10.1007/s12311-023-01625-2\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":50706,\"journal\":{\"name\":\"Cerebellum\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.7000,\"publicationDate\":\"2024-08-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11269489/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cerebellum\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s12311-023-01625-2\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/11/13 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"NEUROSCIENCES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cerebellum","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s12311-023-01625-2","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/11/13 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"NEUROSCIENCES","Score":null,"Total":0}
Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers.
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.
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.