Objective Familial hypercholesterolaemia (FH) elevates low-density lipoprotein cholesterol (LDL-C) and increases cardiovascular disease (CVD) risk. This study aimed to provide evidence for the feasibility of conducting a randomised controlled trial to evaluate the efficacy of an intervention designed to improve diet and physical activity in families with FH.
Design A parallel, randomised, waitlist-controlled, feasibility pilot trial.
Setting Three outpatient lipid clinics in the UK.
Participants Families that comprised children (aged 10–18 years) and their parent with genetically diagnosed FH.
Intervention Families were randomised to either 12-week usual care or intervention. The behavioural change intervention aimed to improve dietary, physical activity and sedentary behaviours. It was delivered to families by dietitians initially via a single face-to-face session and then by four telephone or email follow-up sessions.
Outcome measures Feasibility was assessed via measures related to recruitment, retention and intervention fidelity. Postintervention qualitative interviews were conducted to explore intervention acceptability. Behavioural (dietary intake, physical activity and sedentary time) and clinical (blood pressure, body composition and blood lipids) outcomes were collected at baseline and endpoint assessments to evaluate the intervention’s potential benefit.
Results Twenty-one families (38% of those approached) were recruited which comprised 22 children and 17 adults with FH, and 97% of families completed the study. The intervention was implemented with high fidelity and the qualitative data revealed it was well accepted. Between-group differences at the endpoint assessment were indicative of the intervention’s potential for improving diet in children and adults. Evidence for potential benefits on physical activity and sedentary behaviours was less apparent. However, the intervention was associated with improvements in several CVD risk factors including LDL-C, with a within-group mean decrease of 8% (children) and 10% (adults).
Conclusions The study’s recruitment, retention, acceptability and potential efficacy support the development of a definitive trial, subject to identified refinements.
Trial registration number ISRCTN24880714.
- lipid disorders
- nutrition & dietetics
- paediatric endocrinology
- preventive medicine
- qualitative research
This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See: https://creativecommons.org/licenses/by/4.0/.
Statistics from Altmetric.com
Contributors FJK, JPHS, FEL, GB, CW and DJS contributed to the design of the study. FJK and AS analysed and interpreted the qualitative data. FJK, AET, FEL and JPHS analysed and interpreted the quantitative data. FJK and LH devised and conducted the statistical analysis plan for this analysis. FJK prepared the manuscript, which was critically revised by all coauthors. All authors approved the final version.
Funding This study was funded by the NIHR Biomedical Research Centre at University Hospitals Bristol and Weston NHS Foundation Trust and the University of Bristol.
Competing interests None declared.
Patient consent for publication Not required.
Ethics approval The trial received ethical approval from the Cornwall and Plymouth Research Ethics Committee (reference: 18/SW/0121).
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement Data are available upon reasonable request. Data from this study are available on request.
Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.