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The UK Paediatric Familial Hypercholesterolaemia Register: Statin-related safety and 1-year growth data

Humphries, SE; Cooper, J; Dale, P; Ramaswami, U; FH Paediatric Register Steering Group, .; (2018) The UK Paediatric Familial Hypercholesterolaemia Register: Statin-related safety and 1-year growth data. Journal of Clinical Lipidology , 12 (1) pp. 25-32. 10.1016/j.jacl.2017.11.005. Green open access

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Abstract

BACKGROUND: For children with familial hypercholesterolemia (FH), UK guidelines recommend consideration of statin therapy by age 10 years and dietary and lifestyle advice to maintain an ideal body weight. OBJECTIVES: The objective of the study is to use the UK Paediatric Familial Hypercholesterolemia Register to determine: (1) the prevalence of plasma markers of liver toxicity and muscle damage in statin-treated FH children; (2) the prevalence of obesity in FH children compared to the UK general population; and (3) to compare growth rates in statin-treated and nontreated children. METHODS: Differences in registration and 1-year characteristics were compared by Mann-Whitney U tests. Age and gender body mass index percentiles were compared to UK children's growth charts. RESULTS: In 300 children (51% boys, 75% Caucasian, untreated mean [standard deviation] low-density lipoprotein cholesterol 5.50 [1.49] mmol/L), the proportion on statins varied significantly (P < .005) by age group (<5 years = 0%, 5-10 years = 16.7%, 10-15 years = 57.1%, and >15 years = 73.2%). Statin treatment reduced low-density lipoprotein cholesterol by 31% (1.84 [1.43] mmol/L), and no child showed elevated levels of markers of liver toxicity or muscle damage. At registration, 16.9% of the FH children were overweight (>85th percentile) and 11.1% were obese (>95th percentile) vs reported in 21.2% in UK non-FH children. There was no difference in annual growth rate in statin vs no-statin groups (age-adjusted weight increases 3.58 vs 3.53 kg; P = .91, height 4.45 vs 4.60 cm P = .73). CONCLUSIONS: We show no evidence for statin-related safety or growth issues, but many FH children over the age of 10 years are not on statin treatment. Fewer UK children with FH are obese compared to UK non-FH children.

Type: Article
Title: The UK Paediatric Familial Hypercholesterolaemia Register: Statin-related safety and 1-year growth data
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.jacl.2017.11.005
Publisher version: https://doi.org/10.1016/j.jacl.2017.11.005
Language: English
Additional information: Copyright © 2017 National Lipid Association. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
Keywords: Familial hypercholesterolemia, LDL-C levels, Obesity, Overweight, Pediatric
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Cardiovascular Science
URI: https://discovery.ucl.ac.uk/id/eprint/10044205
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