Temporal Dynamics of the Scale for the Assessment and Rating of Ataxia in Spinocerebellar Ataxias.
Temporal Dynamics of the Scale for the Assessment and Rating of Ataxia in Spinocerebellar Ataxias.
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DOI：
10.1002/mds.29255
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发表时间：
2023-01
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共济失调评估和评级量表（SARA）是评估小脑共济失调严重程度的参考临床量表。在即将进行的治疗试验中，需要可靠的临床结果来评估治疗的有效性。目的是精确评估和比较SARA和新的f-SARA的时间动态。我们分析了来自四个队列（EUROSCA，RISCA，CRC‐SCA和SPATAX）的数据，包括1210名参与者和4092次访视。使用有序贝叶斯混合效应模型（Leaspy）评估进展的线性和变异性。我们进行了不同情况下的治疗试验的样本量计算，以提高规模的反应。八个不同的项目中有七个具有非线性进展。大多数项目之间的进展速度不同，最快项目的平均时间从3.5年[3.4; 3.6]（中位数，95%可信区间）增加到11.4年[10.9; 12.0]。总SARA评分呈线性进展，增加1分的平均时间为0.95 [0.92; 0.98]年。在删除最后四个项目并将所有项目从0重新调整为4后，变异性增加，进展较慢，因此在未来的治疗试验中需要更大的样本量。尽管在项目水平上的异质性的时间动态，SARA的全球进展是线性的。改变初始尺度会降低反应性。这些关于量表时间动态的新信息应该有助于设计未来临床试验的结果。版权所有© 2022作者。运动障碍由Wiley Periodicals LLC代表国际帕金森和运动障碍协会出版。
The Scale for the Assessment and Rating of Ataxia (SARA) is the reference clinical scale to assess the severity of cerebellar ataxia. In the context of upcoming therapeutic trials, a reliable clinical outcome is needed to assess the efficiency of treatments. The aim is to precisely assess and compare temporal dynamics of SARA and a new f‐SARA. We analyzed data from four cohorts (EUROSCA, RISCA, CRC‐SCA, and SPATAX) comprising 1210 participants and 4092 visits. The linearity of the progression and the variability were assessed using an ordinal Bayesian mixed‐effect model (Leaspy). We performed sample size calculations for therapeutic trials with different scenarios to improve the responsiveness of the scale. Seven of the eight different items had a nonlinear progression. The speed of progression was different between most of the items, with an average time for a one‐point increase from 3.5 years [3.4; 3.6] (median, 95% credible interval) for the fastest item to 11.4 [10.9; 12.0] years. The total SARA score had a linear progression with an average time for a one‐point increase of 0.95 [0.92; 0.98] years. After removing the four last items and rescaling all items from 0 to 4, variability increased and progression was slower and thus would require a larger sample size in a future therapeutic trial. Despite a heterogeneous temporal dynamics at the item level, the global progression of SARA was linear. Changing the initial scale deteriorates the responsiveness. This new information about the temporal dynamics of the scale should help design the outcome of future clinical trials. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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