Long-term follow-up of children with familial hypercholesterolaemia treated with cholestyramine.

35 children with the heterozygous form of familial hypercholesterolaemia (FH) who were followed for up to 8 years after starting on cholestyramine treatment showed a progressive decrease in compliance with therapy with time, so that only 55% remained on treatment after 6 years and only 48% after 8 years. Long-term compliance was significantly better in those starting treatment before age 10. Plasma-cholesterol was lowered in all children taking cholestyramine: for the group the mean reduction in plasma-cholesterol ranged from 26 to 44% during the years of the study, on a mean cholestyramine dose of around 0.4 g/kg/day. Cholestyramine thus effectively lowers plasma-cholesterol in FH, but for long-term use compliance is a problem in many children.