Introduction Mobility impairment is the leading cause of disability in the UK. Individuals with congenital mobility impairments have unique experiences of health, quality of life and adaptation. Preference-based outcomes measures are often used to help inform decisions about healthcare funding and prioritisation, however the applicability and accuracy of these measures in the context of congenital mobility impairment is unclear. Inaccurate outcome measures could potentially affect the care provided to these patient groups. The aim of this systematic review was to examine the performance of preference-based outcome measures for the measurement of utility values in various forms of congenital mobility impairment. Methods Ten databases were searched, including Science Direct, CINAHL and PubMed. Screening of reference lists and hand-searching were also undertaken. Descriptive and narrative syntheses were conducted to combine and analyse the various findings. Results were grouped by condition. Outcome measure performance indicators were adapted from COSMIN guidance and were grouped into three broad categories: validity, responsiveness and reliability. Screening, data extraction and quality appraisal were carried out by two independent reviewers. Results A total of 31 studies were considered eligible for inclusion in the systematic review. The vast majority of studies related to either cerebral palsy, spina bifida or childhood hydrocephalus. Other relevant conditions included muscular dystrophy, spinal muscular atrophy and congenital clubfoot. The most commonly used preference-based outcome measure was the HUI3. Reporting of performance properties predominantly centred around construct validity, through known group analyses and assessment of convergent validity between comparable measures and different types of respondents. A small number of studies assessed responsiveness, but assessment of reliability was not reported. Increased clinical severity appears to be associated with decreased utility outcomes in congenital mobility impairment, particularly in terms of gross motor function in cerebral palsy and lesion level in spina bifida. However, preference-based measures exhibit limited correlation with various other condition-specific and clinically relevant outcome measures. Conclusion Preference-based measures exhibit important issues and discrepancies relating to validity and responsiveness in the context of congenital mobility impairment, thus care must be taken when utilising these measures in conditions associated with congenital mobility impairments.

中文翻译：

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先天性活动障碍中健康相关生活质量的基于偏好的测量：有效性和反应性的系统评价。

简介 行动障碍是英国残疾的主要原因。患有先天性行动障碍的人在健康、生活质量和适应方面有着独特的体验。基于偏好的结果测量通常用于帮助就医疗保健资金和优先顺序做出决定，但是这些测量在先天性活动障碍背景下的适用性和准确性尚不清楚。不准确的结果测量可能会影响向这些患者群体提供的护理。本系统评价的目的是检查基于偏好的结果测量在测量各种形式的先天性活动障碍的效用值方面的表现。方法检索了10个数据库，包括Science Direct、CINAHL和PubMed。还进行了参考列表的筛选和手动搜索。进行了描述性和叙述性综合，以结合和分析各种发现。结果按条件分组。结果衡量绩效指标改编自 COSMIN 指南，分为三大类：有效性、响应性和可靠性。筛选、数据提取和质量评估由两名独立评审员进行。结果 共有 31 项研究被认为符合纳入系统评价的条件。绝大多数研究与脑瘫、脊柱裂或儿童脑积水有关。其他相关病症包括肌营养不良、脊髓性肌萎缩和先天性马蹄足。最常用的基于偏好的结果测量是 HUI3。绩效属性的报告主要围绕结构效度，通过已知的组分析和可比措施和不同类型受访者之间的收敛效度评估。少数研究评估了反应性，但没有报告可靠性评估。临床严重程度的增加似乎与先天性活动障碍的效用结果降低有关，特别是在脑瘫的粗大运动功能和脊柱裂的病变水平方面。然而，基于偏好的测量与各种其他特定条件和临床相关的结果测量的相关性有限。结论 基于偏好的测量在先天性活动障碍的情况下表现出与有效性和响应性相关的重要问题和差异，