Responsiveness of UMSARS and other clinical measures in a longitudinal structured care clinic for multiple system atrophy,Clinical Autonomic Research

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Responsiveness of UMSARS and other clinical measures in a longitudinal structured care clinic for multiple system atrophy
Clinical Autonomic Research ( IF 3.9 ) Pub Date : 2022-10-01 , DOI: 10.1007/s10286-022-00898-7
Elisabeth P Golden ₁ , Morgan McCreary ₂ , Steven Vernino ₂

Affiliation

Purpose

As understanding of multiple system atrophy (MSA) pathophysiology improves, clinical trials of disease-modifying therapies are starting. Outcome measures responsive to disease progression will be critical, but the United MSA Rating Scale (UMSARS) has limitations. The MSA multidisciplinary clinic at the University of Texas Southwestern is a longitudinal clinic with structured assessments performed at fixed time intervals. The objective of this study was to evaluate the performance of clinical measures in assessing MSA progression over time.

Methods

Data from 73 subjects with clinically diagnosed MSA were analyzed using repeated measures correlation models. Observations were made every 4 months, with up to 3 years of data included for each patient.

Results

UMSARS-I and UMSARS-II correlated positively with the MSA Quality of Life (QOL) scale. The rate of change was 3.12 points per year (ppy) for UMSARS-I and 5.55 ppy for UMSARS-II.

Some individual UMSARS questions contributed more significantly than others to overall UMSARS rate of change. Based on this finding, and using repeated measures correlations between question combinations and QOL, an optimization of UMSARS parts I and II was curated. The amended UMSARS-I included 8 of the 12 subquestions, and the amended UMSARS-II included 10 of the 14 subquestions.

Conclusions

Data from a longitudinal MSA clinic allows better characterization of the performance of UMSARS as a clinical outcome measure. A curated set of UMSARS questions appears more responsive to change and accounts for correlation with QOL, and could be the starting point for an improved MSA outcome measure.

中文翻译：

UMSARS 和其他临床措施在纵向结构化护理诊所对多系统萎缩的反应

目的

随着对多系统萎缩 (MSA) 病理生理学认识的提高，疾病缓解疗法的临床试验正在启动。对疾病进展作出反应的结果测量将是至关重要的，但联合 MSA 评定量表 (UMSARS) 有局限性。德克萨斯大学西南分校的 MSA 多学科诊所是一个纵向诊所，在固定的时间间隔内进行结构化评估。本研究的目的是评估临床措施在评估 MSA 随时间进展方面的性能。

方法

使用重复测量相关模型分析来自 73 名临床诊断为 MSA 的受试者的数据。每 4 个月进行一次观察，每个患者最多包含 3 年的数据。

结果

UMSARS-I 和 UMSARS-II 与 MSA 生活质量 (QOL) 量表呈正相关。UMSARS-I 的变化率为每年 3.12 点 (ppy)，UMSARS-II 为 5.55 ppy。

一些单独的 UMSARS 问题比其他问题对总体 UMSARS 变化率的贡献更大。基于这一发现，并使用问题组合和 QOL 之间的重复测量相关性，策划了 UMSARS 第 I 部分和第 II 部分的优化。修正后的 UMSARS-I 包括 12 个子问题中的 8 个，修正后的 UMSARS-II 包括 14 个子问题中的 10 个。