Department of Neurology, Johns Hopkins School of Medicine, Baltimore, MD, United States.
Division of Biostatistics, Washington University in St. Louis School of Medicine, St. Louis, MO, United States.
Mult Scler Relat Disord. 2019 Apr;29:86-93. doi: 10.1016/j.msard.2019.01.031. Epub 2019 Jan 24.
People with multiple sclerosis (MS) experience symptoms in multiple domains. High-quality patient-reported outcomes (PROs) that assess multiple domains can aid healthcare providers in assessing these symptoms and may support remote disease monitoring. The "SymptoMScreen" PRO correlates with other PROs in MS; however, whether the SymptoMScreen or its component domains are associated with performance-based or clinician-assessed outcomes is unknown.
To validate SymptoMScreen and its domains against performance-based, clinician-assessed measures or other well-validated diagnostic tools.
We recruited participants with MS from a large tertiary care center. At routine clinic visits participants completed the MS performance test (MSPT), which is an iPad-based application that objectively assesses walking speed, manual dexterity, processing speed, and low contrast letter acuity. Expanded Disability Status Scale (EDSS) scores were assessed in a subset. Participants also completed an online SymptoMScreen following clinic visits. We assessed criterion and construct validity by calculating Spearman rank correlations between the 12 SymptoMScreen domains and respective clinical outcomes. We evaluated test-retest reliability using intra-class correlation coefficients [ICC], and internal consistency reliability using Cronbach's alpha.
The 102 participants were predominantly female (78%), of average age [standard deviation]: 47.6 [12.3] years, with an average disease duration: 13.1 [10.0] years); 60 participants completed the SymptoMScreen and EDSS. Composite SymptoMScreen scores were associated with EDSS (r = 0.71; 95% CI 0.54, 0.83). For individual domains, strong correlations were observed between mobility scores and walking speed (r = 0.63; 95% CI: 0.48, 0.75) and hand function scores with manual dexterity (r = 0.52; 95% CI: 0.36, 0.65). More moderate correlations were detected for the cognition domain with processing speed (r=-0.37; 95% CI: -0.53, -0.18) and for the visual function domain with low contrast letter acuity at 2.5% contrast (r=-0.33; 95% CI -0.54, -0.08). Both test-retest and internal consistency reliability measures for overall SymptoMScreen scores were high (ICC: 0.88; 95% CI: 0.80, 0.93; Cronbach's alpha: 0.93; 95% CI: 0.90, 96).
The SymptoMScreen is practical outcome measure whose subscales may provide a valid assessment of corresponding performance-based and clinician-assessed measures among people with MS with mild-to-moderate disability.
多发性硬化症（MS）患者会出现多个领域的症状。高质量的患者报告结局（PROs）可以评估多个领域，有助于医疗保健提供者评估这些症状，并可能支持远程疾病监测。“SymptoMScreen”PRO 与 MS 中的其他 PRO 相关；然而，SymptoMScreen 或其组成领域是否与基于表现的或临床医生评估的结果相关尚不清楚。
验证 SymptoMScreen 及其各领域与基于表现的、临床医生评估的测量或其他经过良好验证的诊断工具的相关性。
我们从一家大型三级护理中心招募了 MS 患者。在常规就诊时，参与者完成了基于 iPad 的 MS 表现测试（MSPT），该测试客观评估了步行速度、手灵巧性、处理速度和低对比度字母视力。在一部分参与者中还评估了扩展残疾状况量表（EDSS）评分。参与者还在就诊后在线完成了 SymptoMScreen。我们通过计算 12 个 SymptoMScreen 领域与相应临床结果之间的 Spearman 秩相关来评估标准和结构有效性。我们使用组内相关系数 [ICC] 评估测试-重测信度，使用 Cronbach's alpha 评估内部一致性信度。
102 名参与者主要为女性（78%），平均年龄[标准差]：47.6[12.3]岁，平均病程：13.1[10.0]年）；60 名参与者完成了 SymptoMScreen 和 EDSS。综合 SymptoMScreen 评分与 EDSS 相关（r=0.71；95%CI：0.54，0.83）。对于个别领域，移动能力评分与步行速度（r=0.63；95%CI：0.48，0.75）和手部功能评分与手灵巧性（r=0.52；95%CI：0.36，0.65）之间存在很强的相关性。认知领域与处理速度（r=-0.37；95%CI：-0.53，-0.18）以及视觉功能领域与低对比度字母视力（2.5%对比度）之间的相关性则更为适中（r=-0.33；95%CI：-0.54，-0.08）。整体 SymptoMScreen 评分的测试-重测和内部一致性可靠性测量值均较高（ICC：0.88；95%CI：0.80，0.93；Cronbach's alpha：0.93；95%CI：0.90，0.96）。
SymptoMScreen 是一种实用的结局测量工具，其分量表可能为轻度至中度残疾的 MS 患者提供基于表现和临床医生评估的对应测量的有效评估。
