Dose-effect relation of cholestryamine in children and young adults with familial hypercholesterolaemia
Farah, J.R.; Kwiterovich, P.O.; Neill, C.A.
Lancet 1(8002): 59-63
1977
ISSN/ISBN: 0140-6736 PMID: 63709 Document Number: 114864
Children and young adults (20) with familial hypercholesterolemia (F.H.), on a diet low in cholesterol and high in polyunsaturated fats, were treated with cholestyramine in a metabolic unit to determine the decrease in plasma cholesterol and low-density lipoprotein (LDL) cholesterol in relation to drug dosage, pretreatment concentrations of cholesterol and LDL cholesterol, and body-weight. When the dose of cholestyramine was increased in 13 patients by 1 g/day up to 16 g/day, given twice daily, cholesterol and LDL cholesterol fell within the normal range in 11 subjects (average dose, 7 g/day), and the response was directly proportional (P < 0.001) to the pretreatment concentrations of cholesterol (r = 0.89) and LDL cholesterol (r = 0.93) but did not correlate with body weight. Plasma total cholesterol and LDL cholesterol continued to fall and concentrations reached a plateau after which additional cholestyramine had no further effect (average dose, 11 g/day). The LDL cholesterol regression line successfully predicted the dose required to reduce LDL cholesterol concentrations in 7 other patients. There was a significant decrease in mean serum-folate in female patients. The minimum effective dose of cholestyramine in young patients with F.H. can be predicted from the pretreatment plasma total and LDL cholesterol and may be given twice daily.