A Systematic Review and Meta-Analysis of Statin Therapy in Children With Familial Hypercholesterolemia

• Published in: Arteriosclerosis, thrombosis, and vascular biology Vol. 27; no. 8; pp. 1803 - 1810
• Main Authors: Avis, H J, Vissers, M N, Stein, E A, Wijburg, F A, Trip, M D, Kastelein, J J.P, Hutten, B A
• Format: Journal Article
• Language: English
• Published: Philadelphia, PA American Heart Association, Inc 01.08.2007; Hagerstown, MD Lippincott
• Subjects: Adolescent; Age Factors; Atherosclerosis (general aspects, experimental research); Biological and medical sciences; Blood and lymphatic vessels; Blood. Blood coagulation. Reticuloendothelial system; Cardiology. Vascular system; Child; Cholesterol, HDL - blood; Cholesterol, HDL - drug effects; Cholesterol, LDL - blood; Cholesterol, LDL - drug effects; Confidence Intervals; Diseases of the peripheral vessels. Diseases of the vena cava. Miscellaneous; Diseases of the skin. Cosmetics; Dose-Response Relationship, Drug; Double-Blind Method; Drug Administration Schedule; Female; Follow-Up Studies; Humans; Hydroxymethylglutaryl-CoA Reductase Inhibitors - administration & dosage; Hyperlipoproteinemia Type II - diagnosis; Hyperlipoproteinemia Type II - drug therapy; Male; Medical sciences; Pharmacology. Drug treatments; Probability; Radiotherapy. Instrumental treatment. Physiotherapy. Reeducation. Rehabilitation, orthophony, crenotherapy. Diet therapy and various other treatments (general aspects); Randomized Controlled Trials as Topic; Risk Assessment; Severity of Illness Index; Sex Factors; Treatment Outcome; Human; meta-analysis; Metabolic diseases; Cardiovascular disease; Lipids; Hyperlipoproteinemia; Statin derivative; Lipoprotein; Cholesterol; Genetic disease; Vascular disease; Hypercholesterolemia; Treatment; children; safety; statins; Atherosclerosis; familial hypercholesterolemia; Dyslipemia; Child; Antilipemic agent
• ISSN: 1079-5642; 1524-4636
• DOI: 10.1161/ATVBAHA.107.145151

OBJECTIVE—Functional and morphological changes of the arterial wall already present in young children with heterozygous familial hypercholesterolemia (HeFH) suggest that treatment should be initiated early in life to prevent premature atherosclerotic cardiovascular disease. The purpose of this study was to assess the efficacy and particularly safety of statin therapy in children with HeFH. METHODS AND RESULTS—We performed a meta-analysis of randomized, double-blind, placebo-controlled trials evaluating statin therapy in children aged 8 to 18 years with HeFH. Six studies (n=798 children) with 12 to 104 weeks of treatment were included. Total cholesterol, LDL cholesterol, and apolipoprotein B were significantly reduced, whereas HDL cholesterol and apolipoprotein A1 were significantly increased by statin therapy. No statistically significant differences were found between statin- and placebo-treated children with respect to the occurrence of adverse events (RR 0.99; 95% CI0.79 to 1.25), sexual development (RR of advancing ≥1 stage in Tanner classification 0.96; 95% CI0.79 to 1.17), muscle toxicity (RR of CK ≥10 times the upper limit of normal [ULN] 1.38; 95% CI0.18 to 10.82), or liver toxicity (RR of ≥3 times the ULN for ASAT 0.98; 95% CI0.23 to 4.26 and for ALAT 2.03; 95% CI0.24 to 16.95). We found a minimal difference in growth in favor of the statin group (0.33 cm; 95% CI0.03 cm to 0.63 cm). CONCLUSION—In addition to the fact that statin treatment is efficacious, our results support the notion that statin treatment in children with HeFH is safe. Thus, even though further studies are required to assess lifelong safety, statin treatment should be considered for all children aged 8 to 18 with HeFH.