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Treatment with probucol of children with familial hypercholesterolaemia
P Sanjurjo1, P Martul, M Sasieta
1Department of Paediatrics, Cruces Children's Hospital, Bilbao, Spain.
Insights
Probucol effectively lowers high cholesterol in children with familial hypercholesterolaemia when added to a low cholesterol diet. Further research is needed due to unknown long-term effects in pediatric patients.
Area of Science:
- Pediatric Endocrinology
- Cardiovascular Pharmacology
Background:
- Familial hypercholesterolaemia is a genetic disorder leading to high cholesterol levels.
- Effective management in children is crucial to prevent early cardiovascular disease.
Purpose of the Study:
- To evaluate the efficacy and safety of probucol in pediatric patients with familial hypercholesterolaemia.
- To assess the impact of probucol on plasma lipid profiles in children.
Main Methods:
- A cohort of nine children (age 2-12 years) with familial hypercholesterolaemia was studied.
- Children were initially treated with a low fat-cholesterol diet, followed by the addition of probucol (10 mg/kg/day).
- Plasma total cholesterol and high-density lipoprotein cholesterol levels were monitored.
Main Results:
- Adding probucol to a low cholesterol diet reduced mean plasma total cholesterol by an additional 17.1% (from 7.17 to 5.92 mmol/l).
- Absolute levels of high-density lipoprotein cholesterol decreased, but the ratio to total cholesterol remained unchanged.
- No adverse side effects were observed during the study period.
Conclusions:
- Probucol demonstrates significant efficacy in reducing plasma total cholesterol in children with familial hypercholesterolaemia.
- Current use should be limited to severe cases due to unknown long-term drug excretion in children.
Abstract:
Nine children with familial hypercholesterolaemia, age range 2 to 12 years, were treated with a low cholesterol diet and probucol (10 mg/kg/day). The year before, the children received, as only treatment, a low fat-cholesterol diet. During this period their mean plasma total cholesterol level fell from 8.2 +/- 1.45 mmol/l to 7.17 +/- 0.84 mmol/l (12.6%). This level was further reduced to 5.92 +/- 0.63 mmol/l (17.1%) after the addition of probucol. Plasma high density lipoprotein cholesterol levels were lowered in absolute terms but not in relation to total cholesterol. No apparent side effects were observed. However, the use of probucol should be restricted for the moment to severe cases of hypercholesterolaemia as the long-term excretion of the drug in children is not yet known.