Theodorsdottir A, Nielsen H H, Ravnborg M H, Illes Z
Department of Neurology, Odense University Hospital, J.B. Winsloewsvej 4, 5000 Odense C, Denmark; OPEN, Odense Patient Data Explorative Network, Odense University Hospital, Odense, J.B. Winsloewsvej 4, 5000 Odense C, Denmark.
Department of Neurology, Odense University Hospital, J.B. Winsloewsvej 4, 5000 Odense C, Denmark; Department of Neurobiology Research, Institute of Molecular Medicine, University of Southern Denmark, J.B. Winsloewsvej 21, st., 5000 Odense C, Denmark; BRIDGE - Brain Research - Inter Disciplinary Guided Excellence, Department of Clinical Research, J.B. Winsloewsvej 19, 3., 5000 Odense C, Denmark.
Mult Scler Relat Disord. 2023 Mar;71:104550. doi: 10.1016/j.msard.2023.104550. Epub 2023 Feb 5.
Patient-reported outcomes (PROs) are increasingly being used as outcomes in secondary progressive multiple sclerosis (SPMS) trials. We examined how PROs reflect disease burden in SPMS.
In this observational prospective study, 65 SPMS patients were examined by five different PROs (Fatigue Scale Motor Cognition (FSMC), Multiple Sclerosis Impact Scale version 2 (MSIS-29v2), 36-Item Short Form Health Survey version 2 (SF-36v2), EQ-5D-5L and Work Productivity and Activity Impairment Questionnaire: Multiple Sclerosis version 2.0 (WPAI:MS)); two different rating scales, Multiple Sclerosis Impairment Scale (MSIS) and Expanded Disability Status Scale (EDSS); functional tests of mobility (Timed-25-Foot Walk (T-25FW), 6-Spot Step Test (6-SST) and (9-Hole Peg Test (9-HPT)); cognitive tests (Symbol Digital Modalities Test (SDMT) and Brief Visuospatial Memory Test-Revised (BVMT-R)); and multimodal Magnetic Resonance Imaging (MRI).
When the PROs were divided into physical and psychological subscores, the PRO physical subscores of FSMC, MSIS-29v2 and SF-36v2 correlated with physical rating scales (EDSS, MSIS) and physical measures of upper (9-HPT) and lower extremity function (T-25FW and 6-SST)) (p = 0.04-0.0001). 9-HPT correlated the least with physical subscores of PROs but showed the strongest correlation with activity impairment (subscore of WPAI:MS). In contrast, psychological PRO subscores of FSMC, MSIS-29v2 and SF-36v2 did not reflect the cognitive outcomes (SDMT and BVMT-R), although the cognitive scores correlated with disease burden indicated by MRI lesion volumes. The psychological PRO subscores did not correlate with fatigue, physical and MRI outcomes either.
Correlation between PRO physical subscores and physical outcomes supports PROs as potentially useful clinical endpoints in SPMS. The results of this study indicate that patients with SPMS highly perceive their mobility on function of their lower extremities, while they perceive their daily activities highly dependent on function of the upper extremities. Psychological subscores of MS specific PROs may be less suitable as surrogate markers for the cognitive status and should be considered as a mental quality of life measurement independent of disease burden.
患者报告结局（PROs）在继发进展型多发性硬化症（SPMS）试验中越来越多地被用作结局指标。我们研究了PROs如何反映SPMS中的疾病负担。
在这项观察性前瞻性研究中，通过五种不同的PROs（疲劳量表运动认知（FSMC）、多发性硬化症影响量表第2版（MSIS-29v2）、36项简短健康调查问卷第2版（SF-36v2）、EQ-5D-5L和工作效率与活动障碍问卷：多发性硬化症第2.0版（WPAI：MS））对65例SPMS患者进行了评估；两种不同的评定量表，即多发性硬化症损伤量表（MSIS）和扩展残疾状态量表（EDSS）；运动功能测试（25英尺计时步行（T-25FW）、6点步测试（6-SST）和9孔插钉测试（9-HPT））；认知测试（符号数字模态测试（SDMT）和简短视觉空间记忆测试修订版（BVMT-R））；以及多模态磁共振成像（MRI）。
当将PROs分为身体和心理子分数时，FSMC、MSIS-29v2和SF-36v2的PRO身体子分数与身体评定量表（EDSS、MSIS）以及上肢（9-HPT）和下肢功能（T-25FW和6-SST）的身体测量指标相关（p = 0.04 - 0.0001）。9-HPT与PROs的身体子分数相关性最小，但与活动障碍（WPAI：MS的子分数）相关性最强。相比之下，FSMC、MSIS-29v2和SF-36v2的心理PRO子分数并未反映认知结局（SDMT和BVMT-R），尽管认知分数与MRI病变体积所表明的疾病负担相关。心理PRO子分数也与疲劳、身体和MRI结局无关。
PRO身体子分数与身体结局之间的相关性支持PROs作为SPMS中潜在有用的临床终点。本研究结果表明，SPMS患者高度感知其下肢功能对活动能力的影响，而他们认为其日常活动高度依赖上肢功能。特定于MS的PROs的心理子分数可能不太适合作为认知状态的替代标志物，应被视为独立于疾病负担的生活质量心理测量指标。
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