A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

Citations of this article
Mendeley users who have this article in their library.


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

Register to see more suggestions

Mendeley helps you to discover research relevant for your work.

Already have an account?

Save time finding and organizing research with Mendeley

Sign up for free