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Double blind trial of bezafibrate in familial hypercholesterolaemia
Insights
Bezafibrate significantly lowered total cholesterol by 22% in children with familial hypercholesterolaemia. This treatment also moderately increased high-density lipoprotein cholesterol, suggesting its potential as an adjunctive therapy.
Area of Science:
- Pediatric Cardiology
- Clinical Pharmacology
- Lipid Metabolism
Background:
- Familial hypercholesterolaemia (FH) is a genetic disorder leading to high cholesterol levels.
- Early coronary heart disease is a significant risk in children with FH.
- Effective management strategies for pediatric FH are crucial.
Purpose of the Study:
- To evaluate the efficacy of bezafibrate in managing lipid profiles in children with FH.
- To assess the impact of bezafibrate on total cholesterol and high-density lipoprotein cholesterol.
Main Methods:
- A six-month, double-blind, crossover controlled trial.
- Involved 14 children diagnosed with familial hypercholesterolaemia.
- Bezafibrate administered twice daily at 10-20 mg/kg/day, compared against a placebo period.
Main Results:
- Mean plasma total cholesterol concentration was reduced by 22% during bezafibrate treatment compared to placebo.
- A moderate increase in high-density lipoprotein cholesterol was observed.
- No adverse events were detailed in the abstract.
Conclusions:
- Bezafibrate demonstrated significant efficacy in reducing total cholesterol in pediatric FH patients.
- The drug may serve as a valuable adjunctive treatment option for children with familial hypercholesterolaemia.
- Further research may explore long-term outcomes and safety profiles.
Abstract:
A six month, double blind, crossover controlled trial of bezafibrate was conducted in 14 children with familial hypercholesterolaemia all of whom had a strong family history of early coronary heart disease. The bezafibrate was given twice daily in a dose of 10 to 20 mg/kg/day. The mean plasma total cholesterol concentration on bezafibrate was 22% lower than during the period on placebo and there was a moderate rise in high density lipoprotein cholesterol. Bezafibrate may be a useful adjunct to treatment in these children.