An Item Response Theory analysis of the Unified Multiple System Atrophy Rating Scale
An Item Response Theory analysis of the Unified Multiple System Atrophy Rating Scale
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DOI：
10.1016/j.parkreldis.2021.11.024
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发表时间：
2021-12-05
影响因子：
4.1
通讯作者：
Meissner, Wassilios G.
中科院分区：
文献类型：
--
作者：
Foubert-Samier, Alexandra;Pavy-Le Traon, Anne;Meissner, Wassilios G.
简介：统一多系统萎缩评定量表（UMSARS）有四个子量表是专门为MSA患者的临床评估设计的。UMSARS I（日常生活活动）和II（运动检查）亚量表通常被用作治疗试验的主要终点。本研究的主要目的是确定UMSARS I和II子量表项目最能描述随时间的进展。方法：2007年至2020年在法国MSA参考中心就诊的所有MSA患者纳入前瞻性队列，每年进行包括UMSARS在内的随访评估。使用纵向项目反应理论模型对26个UMSARS I和II项目的重复测量进行了分析，以确定UMSARS IV疾病五个阶段中每个阶段信息最丰富的项目。将信息量最大的项目作为一组进一步计算样本量估计值。结果：共纳入557例MSA患者，平均随访2.3年。除了与自主神经异常相关的项目外，大多数项目随着疾病持续时间或在不同的UMSARS IV残疾阶段进展。大约70%的量表信息仅由11/26个项目携带，其中许多反映了患者的观点。这些产生的样本量估计值与非典型肺炎I + II项目相似。结论：本研究为UMSARS I和II亚量表的发展提供了重要信息。对于那些评估自主神经异常的患者，改善似乎特别必要。缩短的量表可能对未来的临床试验结果有用。
Introduction: The Unified Multiple System Atrophy Rating Scale (UMSARS) has four subscales that have been specifically designed for the clinical assessment of MSA patients. UMSARS I (activities of daily living) and II (motor examination) subscales are regularly used as primary endpoints in treatment trials. The main objective of this study was to identify UMSARS I and II subscale items that best describe progression over time.Methods: All MSA patients seen at the French Reference Centre for MSA from 2007 to 2020 were included in a prospective cohort with an annual follow-up assessment including UMSARS. The repeated measures of the 26 UMSARS I and II items were analyzed using a longitudinal Item Response Theory model to identify the most informative items for each of the five UMSARS IV disease stages. Sample size estimates were further calculated for the most informative items as a group.Results: A total of 557 MSA patients were included with a mean follow-up of 2.3 years. The majority of items progressed with disease duration or across the different UMSARS IV disability stages, with the exception of those related to dysautonomia. Roughly 70% of the scale information was carried by only 11/26 items, many reflecting the patient perspective. These yielded similar sample size estimates than UMSARS I + II items.Conclusion: This study provides important information about the progression of UMSARS I and II subscale items. Improvements seem particularly necessary regarding those assessing dysautonomia. A shortened scale may be useful as outcome for future clinical trials.