Dataset Information

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

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.

SUBMITTER: Humphries SE

PROVIDER: S-EPMC5821682 | biostudies-literature | 2018 Jan - Feb

REPOSITORIES: biostudies-literature

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

Humphries Steve E SE Cooper Jackie J Dale Peter P Ramaswami Uma U

Journal of clinical lipidology 20171122 1

<h4>Background</h4>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.<h4>Objectives</h4>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 t ...[more]

PMID: 29208363

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Project description:BackgroundCardiovascular outcomes for people with familial hypercholesterolaemia can be improved with diagnosis and medical management. However, 90% of individuals with familial hypercholesterolaemia remain undiagnosed in the USA. We aimed to accelerate early diagnosis and timely intervention for more than 1·3 million undiagnosed individuals with familial hypercholesterolaemia at high risk for early heart attacks and strokes by applying machine learning to large health-care encounter datasets.MethodsWe trained the FIND FH machine learning model using deidentified health-care encounter data, including procedure and diagnostic codes, prescriptions, and laboratory findings, from 939 clinically diagnosed individuals with familial hypercholesterolaemia (395 of whom had a molecular diagnosis) and 83 136 individuals presumed free of familial hypercholesterolaemia, sampled from four US institutions. The model was then applied to a national health-care encounter database (170 million individuals) and an integrated health-care delivery system dataset (174 000 individuals). Individuals used in model training and those evaluated by the model were required to have at least one cardiovascular disease risk factor (eg, hypertension, hypercholesterolaemia, or hyperlipidemia). A Health Insurance Portability and Accountability Act of 1996-compliant programme was developed to allow providers to receive identification of individuals likely to have familial hypercholesterolaemia in their practice.FindingsUsing a model with a measured precision (positive predictive value) of 0·85, recall (sensitivity) of 0·45, area under the precision-recall curve of 0·55, and area under the receiver operating characteristic curve of 0·89, we flagged 1 331 759 of 170 416 201 patients in the national database and 866 of 173 733 individuals in the health-care delivery system dataset as likely to have familial hypercholesterolaemia. Familial hypercholesterolaemia experts reviewed a sample of flagged individuals (45 from the national database and 103 from the health-care delivery system dataset) and applied clinical familial hypercholesterolaemia diagnostic criteria. Of those reviewed, 87% (95% Cl 73-100) in the national database and 77% (68-86) in the health-care delivery system dataset were categorised as having a high enough clinical suspicion of familial hypercholesterolaemia to warrant guideline-based clinical evaluation and treatment.InterpretationThe FIND FH model successfully scans large, diverse, and disparate health-care encounter databases to identify individuals with familial hypercholesterolaemia.FundingThe FH Foundation funded this study. Support was received from Amgen, Sanofi, and Regeneron.

Dataset Information

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

Publications

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

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