TY - JOUR
T1 - Quantitative Gait and Balance Outcomes for Ataxia Trials
T2 - Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers
AU - Ilg, Winfried
AU - Milne, Sarah
AU - Schmitz-Hübsch, Tanja
AU - Alcock, Lisa
AU - Beichert, Lukas
AU - Bertini, Enrico
AU - Mohamed Ibrahim, Norlinah
AU - Dawes, Helen
AU - Gomez, Christopher M.
AU - Hanagasi, Hasmet
AU - Kinnunen, Kirsi M.
AU - Minnerop, Martina
AU - Németh, Andrea H.
AU - Newman, Jane
AU - Ng, Yi Shiau
AU - Rentz, Clara
AU - Samanci, Bedia
AU - Shah, Vrutangkumar V.
AU - Summa, Susanna
AU - Vasco, Gessica
AU - McNames, James
AU - Horak, Fay B.
N1 - Publisher Copyright:
© 2023, The Author(s).
PY - 2024
Y1 - 2024
N2 - 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.
AB - 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.
KW - Cerebellar ataxia
KW - Digital motor performance marker
KW - Gait and posture
UR - http://www.scopus.com/inward/record.url?scp=85176468332&partnerID=8YFLogxK
U2 - 10.1007/s12311-023-01625-2
DO - 10.1007/s12311-023-01625-2
M3 - Review Article
C2 - 37955812
AN - SCOPUS:85176468332
SN - 1473-4222
VL - 23
SP - 1566
EP - 1592
JO - The Cerebellum
JF - The Cerebellum
ER -