Background The efficacy and safety of atorvastatin in children/adolescents aged 10–17 years with heterozygous familial hypercholesterolemia (HeFH) have been demonstrated in trials of up to 1 year in duration. However, the efficacy/safety of >1 year use of atorvastatin in children/adolescents with HeFH, including children from 6 years of age, has not been assessed. Objective To characterize the efficacy and safety of atorvastatin over 3 years and to assess the impact on growth and development in children aged 6–15 years with HeFH. Methods A total of 272 subjects aged 6–15 years with HeFH and low-density lipoprotein cholesterol (LDL-C) ≥4.0 mmol/L (154 mg/dL) were enrolled in a 3-year study (NCT00827606). Subjects were initiated on atorvastatin (5 mg or 10 mg) with doses increased to up to 80 mg based on LDL-C levels. Results Mean percentage reductions from baseline in LDL-C at 36 months/early termination were 43.8% for subjects at Tanner stage (TS) 1 and 39.9% for TS ≥2. There was no evidence of variations in the lipid-lowering efficacy of atorvastatin between the TS groups analyzed (1 vs ≥2) or in subjects aged <10 vs ≥10 years, and the treatment had no adverse effect on growth or maturation. Atorvastatin had a favorable safety and tolerability profile, and only 6 (2.2%) subjects discontinued because of adverse events. Conclusions Atorvastatin over 3 years was efficacious, had no impact on growth/maturation, and was well tolerated in children and adolescents with HeFH aged 6–15 years.
Langslet, G., Breazna, A., & Drogari, E. (2016). A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia. Journal of Clinical Lipidology, 10(5), 1153-1162.e3. https://doi.org/10.1016/j.jacl.2016.05.010