Acceptability and compliance with two forms of cholestyramine in the treatment of hypercholesterolemia in children: A randomized, crossover trial

Objective: To compare the acceptability, compliance, and effectiveness of two forms of cholestyramine resin in the treatment of hypercholesterolemia in children. Study design: Patients aged 10 to 18 years with familial hypercholesterolemia were enrolled in a randomized, crossover trial of two 8-week periods of either a pill or powder form of cholestyramine at a dose of 8 gm/day. Results: Of 40 children enrolled, 38 completed both medication periods, with a median age of 13 years (range, 10 to 18). At the end of the study, 82% preferred the pill form, 16% the powder form and 2% neither form. Mean (+/-SD) compliance as assessed by the amount of medication taken was significantly greater for pills (61% +/- 31%) than powder (50% +/- 30%, p = 0.01). The form of the medication increased compliance by at least 25% for 16 patients (42%), 13 in favor of pills and 3 in favor of powder. Compliance was not associated with patient attitudes and perceptions of hypercholesterolemia, demographics, family history, previous experience with lipid-lowering medication, or lipid profile parameters. Significant mean reductions in low-density lipoprotein cholesterol concentrations were noted for both pills (-10% +/- 20%, p = 0.006) and powder (-15% +/- 17%, p = 0.0001), with no significant difference between forms (p = 0.16). Conclusions: A change in bile acid-binding resin formulation from powder to pills significantly increases acceptability and compliance in some children with hypercholesterolemia.