Familial hypercholesterolaemia (FH) is a severe disorder of lipid metabolism associated with an enhanced risk to develop cardiovascular disease later in life, with atherosclerotic lesions beginning already in childhood. These are facts which make an early diagnosis and therapy necessary to prevent or delay such complications. The aim of this study was to investigate the efficacy and safety of low-dose simvastatin, a potent HMG-CoA reductase inhibitor, in children and adolescents with FH. Therefore, 20 children and adolescents (12 females, 8 males) aged between 10 and 17 years with FH were recruited for this 1-year simvastatin study. According to baseline levels of low density lipoprotein (LDL)-C, girls and boys were divided into two groups, one group (with LDL-C <220 mg/dl) starting with a simvastatin dosage of 5 mg/day, the other (with LDL-C >220 mg/dl) 10 mg/day with the possibility to increase dosages up to a daily maximum of 20 mg, if not reaching LDL-C concentrations of <170 mg/dl within the first period. Every 4-8 weeks, weight, height, lipids, Lp(a) and routine safety parameters of all participants were determined by a paediatrician, documenting exactly all side-effects. The percentage decrease was 25% for LDL-C in the 5 mg simvastatin period (19% for total cholesterol (tChol)), 30% for LDL-C in the 10 mg period (26% for tChol) and 36% decrease for LDL-C in the 20 mg period (29% for tChol). The changes for high density lipoprotein (HDL)-C were -5.9% (5 mg), +2.9% (10 mg) and -10.9% (20 mg) the percentage decrease in triglycerides was 12.6% (5 mg), 14.3% (10 mg) and 21% (20 mg). The side-effects of simvastatin were of no clinical relevance and all disappeared after a couple of days.
Conclusion: our results showed that simvastatin seems to be an effective and safe medical therapy even in children and adolescents with familial hypercholesterolaemia.