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

J Clin Lipidol. 2016 Sep-Oct;10(5):1153-1162.e3. doi: 10.1016/j.jacl.2016.05.010. Epub 2016 Jun 7.


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.

Keywords: Adolescents; Atorvastatin; Children; Heterozygous familial hypercholesterolemia; Low-density lipoprotein cholesterol; Tanner stage.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Anticholesteremic Agents / adverse effects
  • Anticholesteremic Agents / therapeutic use*
  • Apolipoprotein B-100 / genetics
  • Atorvastatin / adverse effects
  • Atorvastatin / therapeutic use*
  • Child
  • Cholesterol, LDL / blood
  • Female
  • Heterozygote
  • Humans
  • Hyperlipoproteinemia Type II / drug therapy*
  • Hyperlipoproteinemia Type II / pathology
  • Hyperplasia / etiology
  • Male
  • Receptors, LDL / genetics
  • Sarcoma, Ewing / etiology
  • Sex Characteristics


  • Anticholesteremic Agents
  • Apolipoprotein B-100
  • Cholesterol, LDL
  • Receptors, LDL
  • Atorvastatin

Associated data

  • ClinicalTrials.gov/NCT00827606