@article{420221,
author = "Winfried Ilg and Bj{\"o}rn M{\"u}ller and Jennifer Faber and Judith van Gaalen and Holger  Hengel and Ina R. Vogt and Guido Hennes and Bart van de Warrenburg and Thomas  Klockgether and Ludger Sch{\"o}ls and Matthis Synofzik",
abstract = "Measures of step variability and body sway during gait have shown to correlate with clinical ataxia
severity in several cross-sectional studies. However, to serve as a valid progression biomarker,
these gait measures have to prove their sensitivity to robustly capture longitudinal change, ideally
within short time-frames (e.g. one year). We present the first multi-center longitudinal gait analysis
study in spinocerebellar ataxias (SCAs). We performed a combined cross-sectional (n=28) and
longitudinal (1-year interval, n=17) analysis in SCA3 subjects (including 7 pre-ataxic mutation
carriers). Longitudinal analysis revealed significant change in gait measures between baseline and
1-year follow-up, with high effect sizes (stride length variability: p=0.01, effect size rprb=0.66;
lateral sway: p=0.007, rprb=0.73). Sample size estimation for lateral sway reveals a required cohort
size of n=43 for detecting a 50% reduction of natural progression, compared to n=240 for the
clinical ataxia score SARA. These measures thus present promising motor biomarkers for
upcoming interventional studies. ",
journal = "Movement Disorders 2022",
title = "{D}igital gait biomarkers, but not clinical ataxia scores, allow to capture 1-year longitudinal change in {S}pinocerebellar ataxia type 3 ({SCA}3)",
url = "https://www.medrxiv.org/content/10.1101/2022.03.10.22272119v1",
year = "2022",
files = "sca3-2022.03.10.22272119v1.full.pdf",
}