Section Computational Sensomotorics, Hertie Institute for Clinical Brain Research, Tübingen, Germany.
German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany.
Mov Disord. 2022 Nov;37(11):2295-2301. doi: 10.1002/mds.29206. Epub 2022 Aug 31.
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 (eg, 1 year). We present the first multicenter longitudinal gait analysis study in spinocerebellar ataxias. We performed a combined cross-sectional (n = 28) and longitudinal (1-year interval, n = 17) analysis in Spinocerebellar Ataxia type 3 subjects (including seven preataxic mutation carriers). Longitudinal analysis showed significant change in gait measures between baseline and 1-year follow-up, with high effect sizes (stride length variability: P = 0.01, effect size r = 0.66; lateral sway: P = 0.007, r = 0.73). Sample size estimation for lateral sway indicates a required cohort size of n = 43 for detecting a 50% reduction of natural progression, compared with n = 240 for the clinical ataxia score Scale for the Assessment and Rating of Ataxia (SARA). These measures thus present promising motor biomarkers for upcoming interventional studies. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
在几项横断面研究中，步态的步幅变化和身体摆动测量与临床共济失调严重程度相关。然而，作为有效的进展生物标志物，这些步态测量必须证明其能够灵敏地捕捉到纵向变化，理想情况下在较短的时间框架内（例如，1 年）。我们报告了首例多中心脊髓小脑性共济失调的纵向步态分析研究。我们对脊髓小脑性共济失调 3 型患者（包括 7 名未发病的突变携带者）进行了横断面（n=28）和纵向（1 年间隔，n=17）联合分析。纵向分析显示，在基线和 1 年随访之间，步态测量值发生了显著变化，具有较大的效应量（步长变异：P=0.01，效应量 r=0.66；侧向摆动：P=0.007，r=0.73）。侧向摆动的样本量估计表明，与临床共济失调评分量表（SARA）相比，需要 n=43 的队列大小才能检测到自然进展减少 50%，而 n=240 才能检测到侧向摆动。这些措施因此为即将进行的干预研究提供了有前途的运动生物标志物。© 2022 作者。运动障碍由 Wiley 期刊公司代表国际帕金森病和运动障碍协会出版。
