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患者确定的疾病阶段尺度不能与轻度至中度多发性硬化症的扩展疾病状况尺度互换

Yi Chao Foong^1,2,3,4, Daniel Merlo^1,3, Melissa Gresle^1,2,5

¹Department of Neuroscience, Central Clinical School, Monash University, Melbourne, Victoria, Australia.

European journal of neurology

|August 16, 2023

概括

患者确定的疾病阶段 (PDDS) 尺度在多发性硬化症 (MS) 患者中显示出良好的可靠性,但随着时间的推移与扩展残疾状况尺度 (EDSS) 并没有很好的相关性. 对于评估MS进展而言,PDDS不能与EDSS互换.

科学领域:

神经学神经学
临床研究临床研究
患者报告的结果

背景情况:

患者确定的疾病阶段 (PDDS) 尺度的有效性和可靠性在患有轻度至中度残疾的多发性硬化症 (MS) 患者中尚不清楚.
评估MS的疾病进展对于治疗决策至关重要,需要可靠的结局措施.

研究的目的:

在MS患者中检查PDDS尺度的心理测量特性,包括有效性和测试-重新测试可靠性.
评估PDDS尺度的纵向性能及其与扩展残疾状况尺度 (EDSS) 的相关性.

主要方法:

包括复发性复发性MS患者,EDSS得分<4.
评估了PDDS的有效性和测试-重新测试可靠性.
分析了使用混合效应建模和科恩卡帕确认残疾进展 (CDP) 的纵向数据.

主要成果:

PDDS与EDSS的基线相关性很弱 (ρ=0.45),但与患者报告结果 (PRO) 的相关性更强.
在PDDS中,测试重复测试的可靠性很好或很好 (CCC=0.73-0.89).
从长度上看,PDDS与EDSS,年龄和抑郁症的相关性很弱,PDDS和EDSS之间的CDP没有一致性.

结论:

PDDS与PROs的相关性比EDSS更好,但与其他MS结果指标的相关性有限.
对于评估多发性硬化症残疾和进展而言,PDDS和EDSS是不可互换的.
PDDS尺度可能提供一个有价值的以患者为中心的视角,但不应该取代已建立的临床措施,如EDSS.

关键词:

扩展的残疾状况规模.患者决定的疾病步骤多发性硬化症多发性硬化症患者报告的结果.的有效性有效性.

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