MG-ADL 项目和 MG-ADL 分数的心理计量特性:评估两个大型数据集的分布特征、有效性和因子结构。

IF 3.6 3区 医学 Q1 CLINICAL NEUROLOGY Journal of the Neurological Sciences Pub Date : 2024-07-22 DOI:10.1016/j.jns.2024.123135
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引用次数: 0

摘要

背景:重症肌无力-日常生活活动量表(MG-ADL)是一项包含 8 个项目的结果测量,用于评估重症肌无力(MG)患者的症状和功能限制。MG-ADL 评分是一个等权重的水平总分,在临床试验、临床实践中被用作主要结果测量指标,在健康经济评估中也被用作终点。本数据分析旨在详细了解 MG-ADL 项目和 MG-ADL 评分的测量特性:方法:将真实世界前瞻性研究(MRW)的横断面数据与 ADAPT 试验的纵向数据相结合。结果测量包括 MG-ADL、肌无力定量评分(QMG)、MG 15 项生活质量(MG-QOL15r)和 EQ-5D-5L。患者按照美国肌无力基金会(MGFA)的临床分类进行分类。对以下测量特性进行了评估:分布特征、项目间相关性、收敛性、已知组别和构造效度以及内部因素结构:结果:MG-ADL 维度内各项目之间的相关性适中,而 MG-ADL 与可比 MG-QOL15r 和 QMG 项目之间的相关性参差不齐。在 MGFA 类别中,MG-ADL 评分的已知组别有效性得到了证实。按 MGFA 等级划分的 MG-ADL 项目水平平均分显示了构建有效性。包括所有四项结果测量在内的 PCA 得出了九个因子解:讨论:单个 MG-ADL 项目的心理计量特性为中等至良好。本研究表明,MG-ADL 充分体现了 MG 的多维异质性。不过,MG-ADL 评分的心理测量性能参差不齐,这可能会使健康经济建模变得复杂:MyRealWorld-MG于2019年11月25日注册,注册号为NCT04176211。ADAPT随机临床试验已在ClinicalTrials.gov(NCT03669588)注册。
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Psychometric properties of MG-ADL items and MG-ADL score: An assessment of distributional characteristics, validity and factor structure in two large datasets

Background

The Myasthenia Gravis–Activities of Daily Living scale (MG-ADL) is an 8-item outcome measure to assess symptoms and functional limitations in myasthenia gravis (MG) patients. The MG-ADL score is an equally weighted level sum score that is used as primary outcome measures in clinical trials, in clinical practice, and as an end-point in health economic evaluation. This data analysis aims to obtain detailed knowledge of measurement properties of MG-ADL items and the MG-ADL score.

Methods

Cross-sectional data from a real-world prospective study (MRW) were combined with longitudinal data from the ADAPT trial. Outcome measures included were MG-ADL, Quantitative Myasthenia Gravis score (QMG), MG 15-item Quality of Life (MG-QOL15r) and EQ-5D-5L. Patients were categorized by their Myasthenia Gravis Foundation of America (MGFA) clinical classification. The following measurement properties were assessed: distributional characteristics, inter-item correlation, convergent, known groups and construct validity and internal factor structure.

Results

Correlations of items within MG-ADL dimensions were moderate, while MG-ADL correlations between comparable MG-QOL15r and QMG items were mixed. Known groups validity for the MG-ADL score was demonstrated for MGFA class. Mean MG-ADL item level scores by MGFA class demonstrated construct validity. PCA, including all four outcome measures, resulted in a nine factor solution.

Discussion

Psychometric properties of individual MG-ADL items were moderate to good. This study showed that the MG-ADL adequately captures the multidimensional heterogeneous nature of MG. This is, however, accompanied by mixed psychometric performance of the MG-ADL score, which may complicate health economic modelling.

Registration: MyRealWorld-MG was registered on November 25, 2019, with registration number NCT04176211. The ADAPT randomized clinical trial is registered at ClinicalTrials.gov (NCT03669588).

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来源期刊
Journal of the Neurological Sciences
Journal of the Neurological Sciences 医学-临床神经学
CiteScore
7.60
自引率
2.30%
发文量
313
审稿时长
22 days
期刊介绍: The Journal of the Neurological Sciences provides a medium for the prompt publication of original articles in neurology and neuroscience from around the world. JNS places special emphasis on articles that: 1) provide guidance to clinicians around the world (Best Practices, Global Neurology); 2) report cutting-edge science related to neurology (Basic and Translational Sciences); 3) educate readers about relevant and practical clinical outcomes in neurology (Outcomes Research); and 4) summarize or editorialize the current state of the literature (Reviews, Commentaries, and Editorials). JNS accepts most types of manuscripts for consideration including original research papers, short communications, reviews, book reviews, letters to the Editor, opinions and editorials. Topics considered will be from neurology-related fields that are of interest to practicing physicians around the world. Examples include neuromuscular diseases, demyelination, atrophies, dementia, neoplasms, infections, epilepsies, disturbances of consciousness, stroke and cerebral circulation, growth and development, plasticity and intermediary metabolism.
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