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Factors Predicting Statin Initiation During Childhood in Familial Hypercholesterolemia: Importance of Genetic
Noel Peretti1, Alexandre Vimont2, Emmanuel Mas3
1Hospices Civil de Lyon, Pediatric Hospital Femme Mere Enfant HFME, Department of Pediatric Gastroenterology-Hepatology and Nutrition, Bron, France; Lyon University, Claude Bernard Lyon-1 University, Lyon Est Medical school, Place d'Arsonval, Lyon, France; INSERM, CarMeN laboratory, U1060, Oullins, France.
Insights
Genetic diagnosis in children and parental vascular disease predict statin initiation for heterozygous familial hypercholesterolemia (HeFH). These factors aid in early cardiovascular risk management for pediatric patients.
Area of Science:
- Cardiology
- Genetics
- Pediatrics
Background:
- Heterozygous familial hypercholesterolemia (HeFH) is a genetic condition leading to high cholesterol levels.
- Early intervention is crucial to prevent premature atherosclerotic cardiovascular disease (ASCVD).
- Identifying factors influencing statin therapy initiation in children with HeFH is important for effective management.
Purpose of the Study:
- To determine childhood and parental factors associated with starting statin therapy in children diagnosed with HeFH.
- To explore the role of genetic diagnosis and parental history of ASCVD in this treatment decision.
Main Methods:
- A multicenter cohort study involving 245 HeFH child-parent pairs from the REFERCHOL national register (2014-2020).
- Collection of demographic and clinical data, including parental history of ASCVD and vascular disease indicators.
- Logistic regression analysis to identify predictors of statin treatment in children.
Main Results:
- 58% of the 245 children (mean age 14 years) received statin therapy.
- Childhood factors associated with statin use included genetic diagnosis (OR, 2.5), older age (OR, 4.4), more visits (OR, 2.36), and longer follow-up (OR, 1.3).
- Parental vascular disease was a significant predictor of childhood statin treatment (OR, 2.4).
Conclusions:
- Confirmed HeFH via DNA testing in childhood and parental vascular disease are independently linked to statin initiation in pediatric HeFH patients.
- Genetic confirmation of HeFH in children is a key factor for initiating statin therapy.
- These findings support the use of genetic diagnosis for proactive cardiovascular prevention strategies in children.
Objective:
To identify childhood and parental factors associated with initiation of statin therapy in children with heterozygous familial hypercholesterolemia (HeFH), including underlying genetic diagnosis or parental premature atherosclerotic cardiovascular disease (ASCVD).
Study Design:
This multicenter cohort study included 245 HeFH child-parent pairs from the REFERCHOL national register (2014-2020). Demographic and clinical characteristics at the last visit were collected. Vascular disease in parents was defined as a history of ASCVD, and/or a coronary artery calcium score >100, and/or stenosis of >50% in at least carotid artery. Statistical analyses included descriptive analysis, logistic regression for univariate and multivariate effects of statins, and a sensitivity analysis combining the characteristics of children and parents.
Results:
Among the 245 children in the study cohort, 135 (58%), with a mean age of 14 ± 3 years, were treated with a statin. In multivariable analysis, the predictive childhood factors associated with statin treatment were genetic diagnosis (OR, 2.5; 95% CI, 1.3 to 4.9; P = .01), older age (OR, 4.4; 95% CI, 1.8-10.6; P = .01), more than 2 visits (OR, 2.36; 95% CI, 1.18-4.73; P = .015), and longer duration of follow-up (OR, 1.3; 95% CI, 1.1-1.6; P < .001). The predictive parental factor associated with childhood treatment was the presence of vascular disease (OR, 2.4; 95% CI, 1.0-5.7; P = .04).
Conclusions:
HeFH confirmed by DNA testing during childhood and a history of vascular disease in parents were independently associated with statin treatment in children with HeFH. Genetic diagnosis may be useful for cardiovascular prevention in children.
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