113 - Efficacy and safety of statin use in children and adolescents with familial hypercholesterolaemia: a systematic review and meta-analysis of randomized-controlled trials (ID 1236)
Abstract
Background and Aims
Statins are the mainstay of treatment for patients with familial hypercholesterolaemia (FH). The purpose of this study was to systematically investigate and meta-analyze the best available evidence from randomized-controlled trials (RCTs) regarding the efficacy and safety of statins in children and adolescents with FH.
Methods
A comprehensive search was conducted in PubMed, Scopus and Cochrane, up to July 10, 2019. Data were expressed as mean differences with 95% confidence intervals (CI). The I2 index was employed for heterogeneity.
Results
Ten RCTs were included in the qualitative and nine in the quantitative analysis (1,191 patients, aged 13.3±2.5 years). Compared with placebo, statins led to a mean relative reduction in total cholesterol (TC), LDL-C, triglyceride and apo-B concentrations by -25.5% (95% CI -30.4%, -20.5%; I² 91%), -33.8% (95% CI -40.1%, -27.4%; I² 90%), -8.4% (95% CI -14.8%, -2.03%; I² 26%) and -28.8% (95% CI -33.9%, -23.6%; I² 83%), respectively. HDL-C increased by 3.1% (95% CI 1.1% - 5.2%; I² 0%). The effect on TC, LDL-C and apo-B seems to be potency-dependent. More than half of patients may achieve the LDL-C target with high-intensity statin dose (4-14% with low-to-moderate intensity). Baseline LDL-C concentrations do not seem to predict the lipid-lowering effect of statins. Statins were well-tolerated (no effect on growth or sexual development), with no significant differences in transaminase and creatine kinase levels compared with placebo.
Conclusions
Statins are quite effective in reducing TC, LDL-C, TG and apo-B and increasing HDL-C concentrations in children and adolescents with FH. No safety issues were seen with statin use.
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