Objective: To describe the rationale, design, and baseline data of a study conducted to determine the efficacy, safety, and tolerability of simvastatin in children and adolescents with heterozygous familial hypercholesterolemia (FH). Methods: Patients were recruited from nine lipid clinics worldwide. After a 4-week diet/placebo run-in period, patients were randomized to receive either simvastatin or placebo. Simvastatin was started at 10 mg/day and titrated at 8-week intervals to 20 and then 40 mg/day During a second 24-week extension period, patients continued to receive simvastatin 40 mg or placebo daily according to the original allocation. Results: A total of 173 patients [98 boys (age: 13.2 years), 75 girls (age: 14.5 years)] were included in the study Baseline total cholesterol (TC) and low-density lipoprotein cholesterol (LDL-C) were severely elevated in FHboys and girls compared to G9 healthy non-affected controls who were not part of the study. In FH boys and the male siblings, respectively, mean TC was G.78 ± 1.03 vs. 3.80 ± 0.11 mmol/L (P<0.001), and mean LDL-C was 5.09 ± 0.97 vs. 2.53 ± 0.69 mmol/L (P<0.001). In FH girls and the female siblings, respectively, mean TC was 7.44 ± 1.35 vs. 4.24 ± 0.47 mmol/L (P<0.001) and mean LDL-C was 5.68 ± 1.28 mmol/L vs. 2.44 ± 0.50 mmol/L (P<0.001). Conclusion: This is the first and largest randomized, controlled, long-term clinical study to test the efficacy, safety, and tolerability of a statin in boys and girls with FH. The baseline data suggest that the sample selected for this study is representative of patients with FH.

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