Familial Hypercholesterolemia in Children: Treatment With p-Aminosalicylic Acid

Publisher Website
Google Scholar
Abstract
The effect of p-aminosalicylic acid (PAS) on the content of cholesterol and triglycerides in serum and in three lipoprotein classes was determined in 20 children and adolescents with severe familial hypercholesterolemia. Dietary cholesterol and saturated fats were restricted throughout the study. Patients received 150 mg/kg/day of PAS (as PAS-C) or placebo in a single-blind random assignment pattern with crossover at six months. A composite index of compliance was generated for each patient based on interviews, tacit drug inventories, and tests for PAS metabolites. Mean serum cholesterol levels decreased significantly during drug treatment. Cholesterol levels in low-density lipoproteins (LDL) decreased from 273 to 208 mg/100 ml in eight patients whose lipoproteins were fractionated by ultracentrifugation, whereas levels of cholesterol in high-density lipoproteins were unchanged by PAS. Serum triglyceride levels decreased significantly, suggesting that the decrease in LDL with PAS treatment may result in part from inhibition of secretion of lipoprotein precursors. The effect of PAS was sustained for 24 months. No significant toxicity was observed. The lack of effect of PAS on thyroid stimulating hormone suggests that thyroid hyperplasia will not occur at a dose of 150 mg/kg/day. These studies indicate that PAS combined with diet may be useful in the management of familial hypercholesterolemia in childhood.