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

Autor: Ilg W; Section Computational Sensomotorics, Hertie Institute for Clinical Brain Research, Otfried-Müller-Straße 25, 72076, Tübingen, Germany. winfried.ilg@uni-tuebingen.de.; Centre for Integrative Neuroscience (CIN), Tübingen, Germany. winfried.ilg@uni-tuebingen.de., Milne S; Bruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, VIC, Australia.; Department of Paediatrics, Melbourne University, Melbourne, VIC, Australia.; Physiotherapy Department, Monash Health, Clayton, VIC, Australia.; School of Primary and Allied Health Care, Monash University, Frankston, VIC, Australia., Schmitz-Hübsch T; Experimental and Clinical Research Center, a cooperation of Max-Delbrueck Center for Molecular Medicine and Charité, Universitätsmedizin Berlin, Berlin, Germany.; Neuroscience Clinical Research Center, Charité - Universitätsmedizin Berlin, Berlin, Germany., Alcock L; Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK.; NIHR Newcastle Biomedical Research Centre, Newcastle University, Newcastle upon Tyne, UK., Beichert L; Department of Neurodegenerative Diseases and Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany., Bertini E; Research Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesu' Children's Research Hospital, IRCCS, Rome, Italy., Mohamed Ibrahim N; Faculty of Medicine, Universiti Kebangsaan Malaysia, Kuala Lumpur, Malaysia., Dawes H; NIHR Exeter BRC, College of Medicine and Health, University of Exeter, Exeter, UK., Gomez CM; Department of Neurology, The University of Chicago, Chicago, USA., Hanagasi H; Behavioral Neurology and Movement Disorders Unit, Department of Neurology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey., Kinnunen KM; IXICO, London, UK., Minnerop M; Institute of Neuroscience and Medicine (INM-1)), Research Centre Juelich, Juelich, Germany.; Institute of Clinical Neuroscience and Medical Psychology, Medical Faculty & University Hospital Düsseldorf, Heinrich Heine University Düsseldorf, Düsseldorf, Germany.; Department of Neurology, Center for Movement Disorders and Neuromodulation, Medical Faculty & University Hospital Düsseldorf, Heinrich Heine University Düsseldorf, Düsseldorf, Germany., Németh AH; Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK., Newman J; NIHR Newcastle Biomedical Research Centre, Newcastle University, Newcastle upon Tyne, UK.; Wellcome Centre for Mitochondrial Research, Newcastle University, Newcastle upon Tyne, UK., Ng YS; Wellcome Centre for Mitochondrial Research, Newcastle University, Newcastle upon Tyne, UK., Rentz C; Institute of Neuroscience and Medicine (INM-1)), Research Centre Juelich, Juelich, Germany., Samanci B; Behavioral Neurology and Movement Disorders Unit, Department of Neurology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey., Shah VV; Department of Neurology, Oregon Health & Science University, Portland, OR, USA.; APDM Precision Motion, Clario, Portland, OR, USA., Summa S; Movement Analysis and Robotics Laboratory (MARLab), Neurorehabilitation Unit, Neurological Science and Neurorehabilitation Area, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Vasco G; Movement Analysis and Robotics Laboratory (MARLab), Neurorehabilitation Unit, Neurological Science and Neurorehabilitation Area, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., McNames J; APDM Precision Motion, Clario, Portland, OR, USA.; Department of Electrical and Computer Engineering, Portland State University, Portland, OR, USA., Horak FB; Department of Neurology, Oregon Health & Science University, Portland, OR, USA.; APDM Precision Motion, Clario, Portland, OR, USA.
Jazyk: angličtina
Zdroj: Cerebellum (London, England) [Cerebellum] 2024 Aug; Vol. 23 (4), pp. 1566-1592. Date of Electronic Publication: 2023 Nov 13.
DOI: 10.1007/s12311-023-01625-2
Abstrakt: 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.
(© 2023. The Author(s).)
Databáze: MEDLINE
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