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



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.



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.



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.



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.
Department of Endocrinology, Morbid Obesity and Preventive Medicine, Lipid Clinic, Oslo University Hospital, Oslo, Norway. Electronic address: glangsle@ous-hf.no.
Gisle
G
Langslet
Pfizer Inc, New York, NY, USA.
Andrei
A
Breazna
Metabolic Unit, Choremio Goudi Research Institute, 1st Department of Pediatrics, Medical School University of Athens, Aghia Sofia Children's Hospital, Athens, Greece.
Euridiki
E
Drogari
D000293:Adolescent; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D053299:Apolipoprotein B-100 / Q000235:genetics; D000069059:Atorvastatin / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D008078:Cholesterol, LDL / Q000097:blood; D005260:Female; D006579:Heterozygote; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy* / Q000473:pathology; D006965:Hyperplasia / Q000209:etiology; D008297:Male; D011973:Receptors, LDL / Q000235:genetics; D012512:Sarcoma, Ewing / Q000209:etiology; D012727:Sex Characteristics
D000924:Anticholesteremic Agents; D053299:Apolipoprotein B-100; D008078:Cholesterol, LDL; D011973:Receptors, LDL; D000069059:Atorvastatin
Adolescents; Atorvastatin; Children; Heterozygous familial hypercholesterolemia; Low-density lipoprotein cholesterol; Tanner stage