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Atorvastatin Appears to Be the Safest and Most Efficacious in Pediatric Patients With Familial or Severe Hypercholesterolemia


  • 13 y/o male in clinic with strong family history of premature CHD found to have elevated LDL of 190. What are my options? Among children with hyperlipidemia, is one lipid-lowering agent (e.g., atorvastatin) more effective and safer than the others?

Clinical Bottom Lines

  1. CHD is leading cause of morbidity and mortality in US and risk factors for CV disease are increasing in prevalence in pediatric populations.
  2. Large cohort studies show hyperlipidemia in children and adolescents correlates with CAD in adulthood. Risk factor modification and cholesterol-lowering therapy are being studied to decrease overall morbidity and mortality.
  3. Atorvastatin has been found to be more efficacious, with a greater LDL lowering effect than that seen in previous studies of safety and efficacy with other statins. The safety profile also showed minimal difference between placebo and treatment, similar to previous trials.
  4. Study attempts to show atorvastatin has potentially greater lipid lowering effect with similar safety profile at a lower dose than previous statin trials- in absence of head to head data, Atorvastatin does appear to be better choice when therapy required for hyperlipidemia in children.

Summary of Key Evidence

  1. 187 children ages 10-17 randomized to treatment with 10 mg atorvastatin or placebo for 26 weeks. Study powered adequately to detect 15% difference between two groups, with 140 in treatment arm and 47 in placebo arm. Groups with similar characteristics at baseline, and similar cholesterol profiles.1
  2. End-point analysis showed 40% +/- 3.3 reduction in LDL in treatment group with minimal change in placebo group. 60% of patients in treatment arm reached goal LDL of 130 prior to 26 weeks compared with 0% in placebo arm (Number Needed to Treat =2). Children followed with CBCPD, Comp, Basic, CPK- Fisher's 2 point testing of adverse outcomes revealed similar characteristics between placebo and treatment arm with no statistical difference in adverse outcomes.

Additional Comments

  • Studies done largely in white males - need better ethnic and gender diversity in future studies.
  • The study was funded by the makers of atorvastatin.
  • Long term data still is pending in pediatric populations, particularly related to evidence of decreases in morbidity and mortality in adulthood as a result of early intervention.


  1. McCrindle BW, Ose L, Marais AD. Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial. J Pediatr 2003;143:74-80.
  2. Behrman R et. al. ED. Nelson's Textbook of Pediatrics. 17th edition, 2004: 449-451
  3. Berenson GS et. al. Association between multiple cardiovascular risk factors and atherosclerosis in children and young adults. N Engl J Med 1998; 338:1650-6.
  4. Li S, et. al. Childhood cCardiovascular risk factors and carotid vascular disease in adulthood: The Bogalusa Heart Study. JAMA 2003;290: 2271-6.
  5. Wiegman A, et. al. Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA 2004;292(3):331-7.

CAT Author: Matthew Mischler, MD

CAT Appraisers: Ken Pituch , MD

Date appraised: December 15, 2004

Last updated September 23, 2005
Department of Pediatrics and Communicable Diseases
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