Abstract

Abstract Background Familial hypercholesterolemia (FH) is a genetic disorder that may develop serious vascular damage leading to a high risk of the development of premature of cardiovascular disease (CVD). Purpose The aim of this meta-analysis was to evaluate the efficacy of statins on lipid profile in children patients with diagnosed FH. Methods We systematically searched selected electronic databases from inception until 15th March 2022. Main endpoints were lipid profile: total cholesterol (TC), triglycerides (TG), low density lipoprotein cholesterol (LDL-C), high density lipoprotein cholesterol (HDL-C) and apolipoprotein B (Apo-B), lipoprotein(a) (Lp(a)). Random effects mean reduction were used to evaluate the efficacy of statins. Results Eight randomized controlled trial (RCTs) with a total of 1025 children patients with FH (statins n=529) were included in the meta-analysis. At mean follow-up was 10.4 months. We showed that in the group of children treated with statins, compared to control, higher mean TC reduction −1.95 mmol/L (−75 mg/dL) [95% CI, −1.36 to −2.54; p<0.001], TG reduction −0.13 mmol/L (−11.5 mg/dL) [−0.04 to −0.23; p<0.001], and LDL-C reduction −1.94 mmol/L (−75 mg/dL) [−1.43 to −2.45; p<0.001] were observed with significantly higher increase of the mean HDL-C, 0.37 mmol/L (14.3 mg/dL) [0.04 to 0.69; p=0.03] (Figure 1). In addition, the statins significantly reduced Apo-B [−0.50 g/L; p<0.001] and did not significantly affect Lp(a) [0.13 mg/dL; p=0.87] in statins group (Figure 2). Conclusions The results of our meta-analysis clearly showed that children treated with statin therapy had significantly improved lipid profile with lack of effect on Lp(a). This data might help to improve statin therapy in FH children, which is highly underused worldwide. Funding Acknowledgement Type of funding sources: None.

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