Physicians' Academy for Cardiovascular Education

Statin therapy effective and safe in children and adolescents with FH

Efficacy and safety of statin use in children and adolescents with familial hypercholesterolaemia: a systematic review and meta-analysis of randomized-controlled trials

Literature - Anagnostis P, Vaitsi K, Kleitsioti P et al., - Endocrine 2020. doi: 10.1007/s12020-020-02302-8.

Introduction and methods

Familial hypercholesterolemia (FH) is characterized by elevated LDL-c concentrations and is inherited in an autosomal dominant way [1,2]. Heterozygous FH (HeFH) is characterized by LDL-c concentrations varying between 190 and 500 mg/dL (4.9 – 12.9 mmol/L) and affects ~1:200-1:300 people worldwide. Homozygous FH (HoFH) is rarer (1:160,000 to 1:300,000 individuals), with LDL-c concentrations >500 mg/dL (>12.9 mmol/L) [2]. Prolonged exposure to high LDL-c or other lipids predispose FH patients to an increased risk for CVD from childhood age [1]. Statins are the recommended treatment for children and adults with FH [3-6]. In this study, a systematic review and meta-analysis were performed to evaluate the lipid-lowering efficacy of statins on total cholesterol (TC), LDL-c, triglicerides (TG), HDL-c and apolipoprotein B (apo-B) concentrations in children and adolescents with FH in comparison with placebo. Adverse effects associated with statin use and comparative data concerning dose were also studied.

This study included studies in patients (age range 8-18 years) with FH, according to the Dutch Lipid Clinic Score, the US Make Early Diagnosis to Prevent Early Death or the Simon Broom criteria. The included studies provided extractable data and used statin monotherapy in the intervention group and placebo or diet in the control group. A total of ten randomized clinical trials (RCTs) were included in the qualitative analysis and nine in the quantitative analysis, yielding a total number of 1191 individuals with FH (all with HeFH, mean age was 13.3 ± 2.5 years, ranging from 10.6±2.9 to 15±2 years).

Main results


Statin treatment in children and adolescents with FH reduces TC, LDL-c and apo-B. A modest reduction in TG concentrations and modest increase in HDL-c levels were also observed. Statin potency appears to have an effect on the lipid-lowering efficacy in this age group. Statins were generally well tolerated and no clinical signs of growth or sexual impairment were observed.


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