Efficacy and safety of cholestyramine therapy in peripubertal and prepubertal children with familial hypercholesterolemia.
OBJECTIVE
To determine the efficacy and safety of cholestyramine therapy in young children with familial hypercholesterolemia.


METHODS
Boys aged 6 to 11 years (n = 57) and girls aged 6 to 10 years (n = 39) with familial hypercholesterolemia.


METHODS
After 1 year of a low-fat, low-cholesterol diet, children with low-density lipoprotein (LDL) cholesterol levels > or = 4.9 mmol/L (190 mg/di) or < or = 4.1 mmol/L (160 mg/dl) in the presence of familial premature cardiovascular disease were randomly assigned to a double-blind comparison of 8 gm cholestyramine (n = 36) and placebo (n = 36) for 1 year.


METHODS
The primary efficacy and safety outcomes were serum LDL cholesterol levels and height velocity, respectively. Secondary safety outcomes were erythrocyte folate, total plasma homocysteine, serum fat-soluble vitamins, and side effects.


RESULTS
Twenty-two subjects in the cholestyramine group and 26 in the placebo group completed the 1-year study. Most withdrawals from the study were related to unpalatability of the study drug or placebo. The LDL cholesterol levels changed by -16.9% (95% confidence interval, -10.8% to -22.9%) in the cholestyramine group compared with 1.4% (95% confidence interval, -4.4% to 7.2%) in the placebo group. Mean height velocity standard deviation scores during 1 year for the children in the cholestyramine and the placebo groups who had not started puberty were 0.24 +/- 1.14 and 0.11 +/- 0.68, respectively (not significant). In the cholestyramine group, mean levels of 25-hydroxyvitamin D decreased. One girl had low folate and elevated homocysteine levels, and there was one case of intestinal obstruction caused by adhesions.


CONCLUSIONS
Significant reductions in LDL cholesterol are achievable during treatment with cholestyramine in about half of eligible children. Growth is not adversely affected. Folate deficiency may occur, even with a low dose of cholestyramine, and vitamin D supplements should be considered. Caution should possibly be exercised in starting cholestyramine therapy within 3 months of abdominal surgery in children.
Tonstad
S
S
Lipid Clinic, Medical Department A, National Hospital, Oslo, Norway.
Knudtzon
J
J
Sivertsen
M
M
Refsum
H
H
Ose
L
L
D000924:Anticholesteremic Agents / Q000627:therapeutic use*; D002648:Child; D002792:Cholestyramine Resin / Q000627:therapeutic use*; D004311:Double-Blind Method; D002149:Energy Intake; D005260:Female; D006128:Growth; D006710:Homocysteine / Q000097:blood; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000178:diet therapy / Q000188:drug therapy*; D008297:Male
D000924:Anticholesteremic Agents; D006710:Homocysteine; D002792:Cholestyramine Resin