Abstract 14231: Effectiveness and Safety of Statin Therapy in Children: A Multi-Center, Real-World Clinical Practice Experience

Abstract

Results of statin use in clinical practice in children are very limited, with effectiveness and safety data based on short-term clinical trials. To address this, we reviewed the results of all children with primary hypercholesterolemia treated with statins for more than 6 months from 4 Pediatric Lipid Clinics. Expert Panel guidelines recommend statin if LDL-cholesterol (LDL-C) exceeds 4.9 mmol/L (190 mg/dL), or 4.1 mmol/L (160 mg/dL) with additional risk factors, after 6 months of lifestyle modification, with hepatic enzyme monitoring plus clinical surveillance and CK measurement for myositis. Patients at all study sites were managed using these guidelines. Results: There were 246 pts (59% male) who had 1488 clinical assessments. Mean age at statin initiation was 12.5±0.5 yrs. Mean duration of therapy was 2.9 yrs (IQR: 1.9_4.7) with 48% more than 3 yrs. Initial statin prescribed varied over time but 61% of pts were started on atorvastatin and 70% remained on the first statin prescribed. Mean compliance was assessed at 92% and 234 pts (94%) remained on statin therapy at the end of the review. Baseline and follow-up lab values and anthropometry (mean + SD) appear below. While 13 pts (5%) had transient AST or ALT > 3xULN, or CK >10xULN at some time during follow-up, no pt was diagnosed with myositis. In regression analysis adjusted for repeated measures over time, statin treatment was associated with significant reductions in total cholesterol, LDL-C and non-HDL-C with no change in HDL-C, TG, safety labs or anthropometry. Despite statin, 51% and 70% of pts had LDL-C levels above minimal (<3.35mmol/L; 130 mg/dL) and ideal (<2.85mmol/L; 110 mg/dL) targets at last follow-up. Conclusions: These findings in a large series of pts from real-world clinical practice show that statin therapy in children with primary hypercholesterolemia is safe and effectively lowers LDL-C on mid-term follow-up. Side effects are rare and discontinuation of treatment is uncommon.