Effectiveness and Safety of Statin Therapy in Children: A Real-World Clinical Practice Experience

Abstract

BACKGROUND: Statin use for hypercholesterolemia in children is predominantly reported from short-term clinical trials. In this study, we assess the efficacy and safety of statin treatment in clinical pediatric practice. METHODS: Records of all patients who began statin treatment at age <18 years and remained on statins for >6 months from 5 pediatric lipid clinics were reviewed. Information at baseline and from all clinic evaluations after statin initiation was recorded, including lipid measurements, statin drug/dose, safety measures (anthropometry, hepatic enzymes, creatine kinase levels), and symptoms. Lipid changes on statin therapy were assessed from baseline to 6 ± 3 months and from 6 ± 3 months to last follow-up with a mixed-effects model, using piecewise linear splines to describe temporal changes, controlling for repeated measures, sex, and age. RESULTS: < 0.001). Triglycerides and HDL-C were unchanged but the triglyceride to HDL-C ratio decreased significantly by late follow-up. At final evaluation, median LDL-C had decreased to 3.4 mmol/L (IQR:2.8-4.2). No patient had statins discontinued for safety measures or symptoms. CONCLUSIONS: In real-world clinical practice, intermediate-term statin treatment is effective and safe in children and adolescents with severe LDL-C elevation.