Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial.
OBJECTIVE
To determine the safety and efficacy of atorvastatin (10 to 20 mg) in children and adolescents with familial hypercholesterolemia or severe hypercholesterolemia.


METHODS
Subjects (n=187) were randomly assigned to 26 weeks of treatment with atorvastatin (10 mg) or placebo. Dosage was increased to 20 mg if LDL cholesterol (LDL-C) levels remained >3.4 mmol/L (130 mg/dL) at week 4. At week 26, subjects received 10 mg of atorvastatin for an additional 26 weeks. Efficacy variables included percent changes in LDL-C, total cholesterol, triglycerides, HDL cholesterol, and apolipoprotein B from baseline to week 26.


RESULTS
Atorvastatin caused a highly significant reduction in LDL-C compared with placebo (-40% vs -0.4%, respectively; P<.001). Percent changes at week 26 also significantly favored atorvastatin for total cholesterol (-32% vs -1.5%; P<.001), triglycerides (-12% vs +1.0%; P=0.03), and apolipoprotein B (-34% vs +0.7%; P<.001), with a significantly greater increase in HDL cholesterol with atorvastatin compared with placebo (+2.8% vs -1.8%; P=.02). Atorvastatin was as well-tolerated as placebo.


CONCLUSIONS
Treatment with atorvastatin for 12 months was effective and safe for pediatric subjects with known familial hypercholesterolemia or severe hypercholesterolemia.
McCrindle
Brian W
BW
Division of Pediatric Cardiology, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada. brian.mccrindle@sickkids.ca
Ose
Leiv
L
Marais
A David
AD
D000293:Adolescent; D000328:Adult; D001055:Apolipoproteins B / Q000097:blood; D000069059:Atorvastatin; D002648:Child; D008076:Cholesterol, HDL / Q000097:blood; D008078:Cholesterol, LDL / Q000097:blood; D004311:Double-Blind Method; D005260:Female; D006538:Heptanoic Acids / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D006937:Hypercholesterolemia / Q000097:blood / Q000188:drug therapy / Q000235:genetics; D006949:Hyperlipidemias / Q000097:blood / Q000188:drug therapy* / Q000235:genetics*; D000960:Hypolipidemic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D011758:Pyrroles / Q000009:adverse effects / Q000627:therapeutic use*; D012720:Severity of Illness Index; D014280:Triglycerides / Q000097:blood
D001055:Apolipoproteins B; D008076:Cholesterol, HDL; D008078:Cholesterol, LDL; D006538:Heptanoic Acids; D000960:Hypolipidemic Agents; D011758:Pyrroles; D014280:Triglycerides; D000069059:Atorvastatin