Abstract

19 children with heterozygous familial hypercholesterolaemia have been treated with cholestyramine administered twice daily in a total dosage of 8 to 24 g/day (0·3 to 1·1 g/kg body weight per day). Serum cholesterol concentration was reduced by a mean of 36% (range 27 to 47%). The therapeutic effect was similar whether or not dietary fat was restricted, and the reduction in serum cholesterol has been maintained for periods of up to 20 months.

Side effects of cholestyramine have been confined to the gastrointestinal tract. Absorption of fat was impaired in some patients, but has not been associated with diarrhoea. Serum folate levels have decreased in all patients, and 6 out of 12 tested have had subnormal red blood cell folate. There has been no evidence of malabsorption of other vitamins or of minerals. Growth rate has been normal in all children.