Gisle Langslet1, Andrei Breazna2, Euridiki Drogari3. 1. Department of Endocrinology, Morbid Obesity and Preventive Medicine, Lipid Clinic, Oslo University Hospital, Oslo, Norway. Electronic address: glangsle@ous-hf.no. 2. Pfizer Inc, New York, NY, USA. 3. Metabolic Unit, Choremio Goudi Research Institute, 1st Department of Pediatrics, Medical School University of Athens, Aghia Sofia Children's Hospital, Athens, Greece.
Abstract
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.
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.
Authors: Alpo Vuorio; Jaana Kuoppala; Petri T Kovanen; Steve E Humphries; Serena Tonstad; Albert Wiegman; Euridiki Drogari; Uma Ramaswami Journal: Cochrane Database Syst Rev Date: 2017-07-07
Authors: Alpo Vuorio; Jaana Kuoppala; Petri T Kovanen; Steve E Humphries; Serena Tonstad; Albert Wiegman; Euridiki Drogari; Uma Ramaswami Journal: Cochrane Database Syst Rev Date: 2019-11-07
Authors: Charlotte Gimpel; Carsten Bergmann; Detlef Bockenhauer; Luc Breysem; Melissa A Cadnapaphornchai; Metin Cetiner; Jan Dudley; Francesco Emma; Martin Konrad; Tess Harris; Peter C Harris; Jens König; Max C Liebau; Matko Marlais; Djalila Mekahli; Alison M Metcalfe; Jun Oh; Ronald D Perrone; Manish D Sinha; Andrea Titieni; Roser Torra; Stefanie Weber; Paul J D Winyard; Franz Schaefer Journal: Nat Rev Nephrol Date: 2019-11 Impact factor: 28.314