Details

Autor(en) / Beteiligte

• Carreau, Valerie
• Girardet, Jean-Philippe
• Bruckert, Eric

Titel

Long-Term Follow-Up of Statin Treatment in a Cohort of Children with Familial Hypercholesterolemia

Ist Teil von

• Paediatric drugs, 2011-08, Vol.13 (4), p.267

Ort / Verlag

Auckland: Springer

Erscheinungsjahr

2011

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• Background Early identification of children with familial hypercholesterolemia (FH) makes it possible to start lipid-lowering therapy at a young age in order to prevent cardiovascular disease. Numerous randomized, often placebo-controlled, studies have assessed the efficacy and safety of statins in children with FH. Objective The aim of this pragmatic observational study was to evaluate pravastatin treatment efficacy and tolerability for a long period of time, and to assess how these results translate in 'real-life' clinical practice. Methods We analyzed all medical files of young hypercholesterolemic patients referred to two specialized French centers. This population of 185 pravastatin-treated children with FH, with a mean baseline cholesterol level above 300 mg/dL, in most of whom genetic diagnosis was achieved, was followed-up for a mean duration of 2 years 2 months. The mean age for starting pravastatin was 11 years; in one of five children, treatment was started before the age of 8 years, mostly because of severe hypercholesterolemia or a family history of coronary heart disease. Results A 16.9-19.2% decrease in total cholesterol level (21-24% for low-density lipoprotein cholesterol) was observed. Growth and puberty were not affected by statin treatment. A review of the medical files showed that 13% of children had side effects, most of which were minor; four of these children had muscular symptoms possibly related to the treatment. This frequency is lower than that observed in adults, and comparable to other studies in children. Conclusions In this large cohort of FH children, the efficacy and tolerability of pravastatin therapy in reallife conditions was demonstrated to be similar to that in randomized controlled studies.