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Published on: November 10, 2017
The effect of low-dose simvastatin in children with familial hypercholesterolaemia: a 1-year observation
Albert Dirisamer1, Nilouparak Hachemian, Robert Alexander Bucek
1Department of Paediatrics, University of Vienna, Währinger Gürtel 18-20, 1090, Austria.
Insights
Low-dose simvastatin effectively lowers cholesterol in children and adolescents with familial hypercholesterolaemia (FH). This study found simvastatin to be a safe treatment option for managing FH in pediatric patients.
Area of Science:
- Pediatrics
- Cardiology
- Pharmacology
Background:
- Familial hypercholesterolaemia (FH) is a genetic lipid metabolism disorder.
- FH significantly increases the risk of early-onset cardiovascular disease due to atherosclerotic lesions.
- Early diagnosis and treatment are crucial for preventing cardiovascular complications in FH patients.
Purpose of the Study:
- To evaluate the efficacy and safety of low-dose simvastatin in children and adolescents diagnosed with FH.
- To assess the impact of simvastatin on lipid profiles, including LDL-C, total cholesterol, HDL-C, and triglycerides.
- To monitor for any adverse effects associated with simvastatin treatment in this pediatric population.
Main Methods:
- A 1-year study involving 20 children and adolescents (aged 10-17) with FH.
- Participants were divided into groups receiving initial simvastatin doses of 5 mg/day or 10 mg/day based on baseline LDL-C levels.
- Dosages could be increased up to 20 mg/day if LDL-C targets (<170 mg/dl) were not met; regular monitoring of weight, height, lipids, and safety parameters was conducted.
Main Results:
- Simvastatin treatment led to significant reductions in LDL-C and total cholesterol across all dosage groups (5 mg, 10 mg, 20 mg).
- LDL-C decreased by 25-36%, and total cholesterol by 19-29% depending on the dosage.
- HDL-C and triglyceride levels also showed favorable changes, with triglycerides decreasing by 12.6-21%.
Conclusions:
- Low-dose simvastatin demonstrates effectiveness in managing lipid levels in pediatric patients with FH.
- The study indicates that simvastatin is a safe therapeutic option for children and adolescents with familial hypercholesterolaemia.
- Observed side effects were minor and transient, supporting the favorable safety profile of simvastatin in this age group.
Unlabelled:
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.
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