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

Ilg W 1 , Milne S 2 , Schmitz-Hübsch T 3 , Alcock L 4 , Beichert L 5 , Bertini E 6 Show all authors , Mohamed Ibrahim N 7 , Dawes H 8 , Gomez CM 9 , Hanagasi H 10 , Kinnunen KM 11 , Minnerop M 12 , Németh AH 13 , Newman J 14 , Ng YS 15 , Rentz C 12 , Samanci B 10 , Shah VV 16 , Summa S 17 , Vasco G 17 , McNames J 18 , Horak FB 16 , Ataxia Global Initiative (AGI) working group Digital Motor Biomarkers

Affiliations 

  • 1 Section Computational Sensomotorics, Hertie Institute for Clinical Brain Research, Otfried-Müller-Straße 25, 72076, Tübingen, Germany. winfried.ilg@uni-tuebingen.de
  • 2 Bruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, VIC, Australia
  • 3 Experimental and Clinical Research Center, a cooperation of Max-Delbrueck Center for Molecular Medicine and Charité, Universitätsmedizin Berlin, Berlin, Germany
  • 4 Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK
  • 5 Department of Neurodegenerative Diseases and Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany
  • 6 Research Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesu' Children's Research Hospital, IRCCS, Rome, Italy
  • 7 Faculty of Medicine, Universiti Kebangsaan Malaysia, Kuala Lumpur, Malaysia
  • 8 NIHR Exeter BRC, College of Medicine and Health, University of Exeter, Exeter, UK
  • 9 Department of Neurology, The University of Chicago, Chicago, USA
  • 10 Behavioral Neurology and Movement Disorders Unit, Department of Neurology, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey
  • 11 IXICO, London, UK
  • 12 Institute of Neuroscience and Medicine (INM-1)), Research Centre Juelich, Juelich, Germany
  • 13 Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK
  • 14 NIHR Newcastle Biomedical Research Centre, Newcastle University, Newcastle upon Tyne, UK
  • 15 Wellcome Centre for Mitochondrial Research, Newcastle University, Newcastle upon Tyne, UK
  • 16 Department of Neurology, Oregon Health & Science University, Portland, OR, USA
  • 17 Movement Analysis and Robotics Laboratory (MARLab), Neurorehabilitation Unit, Neurological Science and Neurorehabilitation Area, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy
  • 18 APDM Precision Motion, Clario, Portland, OR, USA
Cerebellum, 2023 Nov 13.
PMID: 37955812 DOI: 10.1007/s12311-023-01625-2

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.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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