期刊
MOVEMENT DISORDERS
卷 38, 期 1, 页码 35-44出版社
WILEY
DOI: 10.1002/mds.29255
关键词
spinocerebellar ataxia; Scale for the Assessment and Rating of Ataxia; disease course mapping
This study analyzed the temporal dynamics of SARA and f-SARA in assessing the severity of cerebellar ataxia. The global progression of SARA was found to be linear, while f-SARA showed slower progression, requiring a larger sample size for therapeutic trials.
Background: 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. Objective: The aim is to precisely assess and compare temporal dynamics of SARA and a new f-SARA. Methods: 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. Results: 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. Conclusion: 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. (c) 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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