Safety and Efficacy of Long-Term Diet and Diet Plus Bile Acid-Binding Resin Cholesterol-Lowering Therapy in 73 Children Heterozygous for Familial Hypercholesterolemia

Our specific aim was to examine the efficacy and safety of long-term cholesterol-lowering diet and bile acid-binding resin therapy in 73 children heterozygous for familial hypercholesterolemia (FH). We prospectively followed accretion of height and weight in 40 FH children for 5.8 years on diet alone and in 33 FH children for 4.3 years on diet and bile acid-binding resins (8 to 20 g/d). In 67 of these 73 children, sequential data on plasma choleterol lowering was obtained, including 32 children on diet plus bile acid-binding resins and 35 on diet alone. For all 73 children, median age, sex, and race-specific percentiles for height and weight at entry were 50 and 50, respectively, and, 5.7 years later, were unchanged at 50 and 50. Initial and final percentiles for height (r = .76, P < .001) and weight (r = .70, P < .001) were closely correlated. Percentile distributions for height and weight at entry into the study did not differ from those at the end of follow-up (P > .1), in both the 40 FH children on diet alone and the 33 on diet plus bile acid-binding resins. Tracking of height and weight did not differ in the 40 children on diet alone v the 33 on diet plus bile acidbinding resins (P > .1). During 6 years of follow-up there were no significant differences in the percentage of serial, postbaseline measurements for height which were either less than or greater than or equal to baseline percentiles, comparing 40 FH children on diet alone, 33 FH children on diet plus resin, and 39 normal children (on ad libitum diet). FH children on diet or plus resin had a smaller percentage of weight measurements equal to or more than baseline percentiles than normals on follow-up (P < .01), probably reflecting restriction of total fat intake to < 35% of calories. On diet alone, 32 FH children had total plasma cholesterol of 307 ± 8 mg/dL (mean ± SE); bile acidbinding resins were added to diet in these children at an average age of 11.5 years, with this regimen maintained for 4.6 ± 0.4 years, leading to a mean reduction in total plasma cholesterol of 12.5% ± 2% beyond the effects of diet alone (P<.01). On diet plus bile acid-binding resins, 44% of children had a reduction of total plasma cholesterol ≤14%; 28% had a reduction ≥21% (beyond the effects of diet alone.) Thirty-five FH children (starting at 11.7 years of age) were treated with diet alone. Their mean total plasma cholesterol at baseline was 243 ± 6 mg/dl and decreased 9.6% ± 2% (P<.01) after an average of 4.5 ± 0.5 years of diet. On diet alone, 29% of children had a reduction of total plasma cholesterol ≥14%, and 23% had a reduction ≥21%. Long-term diet and bile acid-binding resin therapy did not affect normal growth in 73 FH children and was effective in reducing total plasma cholesterol levels within ranges previously shown to have efficacy in reducing coronary heart disease events in hypercholesterolemic men.