Department of Physical Therapy, Faculty of Health Sciences, Universidade Federal dos Vales do Jequitinhonha e Mucuri, Diamantina, Minas Gerais, Brazil.
School of Physical Education, Physiotherapy and Occupational Therapy, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Dev Med Child Neurol. 2022 Dec;64(12):1453-1461. doi: 10.1111/dmcn.15345. Epub 2022 Jul 21.
To identify the standardized assessment scales for people with muscular dystrophy and investigate the quality/level of evidence of their measurement properties.
A systematic review of patient-reported outcome measures was conducted on the MEDLINE, Embase, AMED, DiTA, and PsycINFO databases in August 2020. We included psychometric studies that investigated the validity, reliability, and responsiveness of instruments assessing activity and participation for muscular dystrophy of any type (Duchenne, Becker, limb-girdle, facioscapulohumeral, congenital, and myotonic) or age. Two independent reviewers selected the studies, extracted data, and evaluated the instruments' quality and level of evidence following the COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) checklist. The study followed the Preferred Reporting Items for Systematic reviews and Meta-Analysis (PRISMA) 2020 guidelines.
The searches identified 6675 references; a total of 46 studies with 28 condition-specific or general instruments were included. The measurement properties of most instruments had sufficient (68.8%) or indeterminate (25.7%) results according to COSMIN. The quality of evidence of the measurement properties was moderate (23.8%) or low (22.6%) according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE).
There is a lack of high-quality instruments whose psychometric properties are adequately measured. The highest quality instrument is the Muscular Dystrophy Functional Rating Scale. The Motor Function Measure (general instrument), Duchenne Muscular Dystrophy Upper-limb Patient-reported Outcome Measure, North Star Ambulatory Assessment, and Myotonic Dystrophy Type 1 Activity and Participation Scale for Clinical Use (specific) are also recommended.
There are 28 available instruments for activity and participation of people with muscular dystrophy. The evidence quality is moderate or low because of imprecision and indirectness. The Muscular Dystrophy Functional Rating Scale is the highest quality instrument. The Motor Function Measure is the second most recommended instrument. The Duchenne Muscular Dystrophy Upper-limb Patient-reported Outcome Measure, North Star Ambulatory Assessment, and Myotonic Dystrophy Type 1 Activity and Participation Scale for Clinical Use are also recommended.
确定用于肌营养不良症患者的标准化评估量表，并调查其测量特性的质量/证据水平。
2020 年 8 月，我们对 MEDLINE、Embase、AMED、DiTA 和 PsycINFO 数据库进行了系统的患者报告结局测量评估综述。我们纳入了研究工具的有效性、信度和反应性的心理计量学研究，这些工具评估了任何类型（杜氏、贝克氏、肢带型、面肩肱型、先天性和肌强直性）或年龄的肌营养不良症患者的活动和参与情况。两名独立的综述作者选择研究、提取数据，并根据共识基于标准选择健康状态测量工具（COSMIN）检查表评估工具的质量和证据水平。研究遵循系统评价和荟萃分析的 Preferred Reporting Items（PRISMA）2020 指南。
搜索共确定了 6675 条参考文献；共纳入 46 项研究，涉及 28 种特定疾病或通用工具。根据 COSMIN，大多数工具的测量特性具有足够（68.8%）或不确定（25.7%）的结果。根据推荐评估、制定和评价（GRADE），测量特性的证据质量为中等（23.8%）或低（22.6%）。
缺乏经过充分测量其心理计量特性的高质量工具。质量最高的工具是肌营养不良功能评定量表。还推荐使用通用工具运动功能测定法、杜氏肌营养不良上肢患者报告结局测量、北极星动态评估和用于临床使用的肌强直性肌营养不良 1 型活动和参与量表。
有 28 种用于肌营养不良症患者活动和参与的工具。由于不精确和间接性，证据质量为中等或低。肌营养不良功能评定量表是质量最高的工具。运动功能测定法是第二推荐的工具。杜氏肌营养不良上肢患者报告结局测量、北极星动态评估和用于临床使用的肌强直性肌营养不良 1 型活动和参与量表也被推荐使用。
