Preference-based measures of health-related quality of life in congenital mobility impairment: a systematic review of validity and responsiveness

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.