Colesevelam: in pediatric patients with heterozygous familial hypercholesterolemia.

Colesevelam hydrochloride (colesevelam), a non-absorbed, synthetic, lipid-lowering polymer, is a bile acid sequestrant. Colesevelam binds with high affinity to bile acids within the gastrointestinal tract, thereby inhibiting the reabsorption of bile acids, resulting in decreases in serum low-density lipoprotein cholesterol (LDL-C) levels. Colesevelam is available as tablets and as powder for oral suspension. At dosages of 3.75 g once daily or 1.875 g twice daily, colesevelam is approved in the US for the treatment of pediatric patients aged 10-17 years with heterozygous familial hypercholesterolemia. Colesevelam may be administered as monotherapy or in combination with an HMG-CoA reductase inhibitor (statin). A 32-week trial was conducted and consisted of a stablilization period ( approximately 4 weeks), a randomized period (8 weeks), an open-label period (18 weeks), and a 2-week follow-up period. In the 8-week, randomized, double-blind, placebo-controlled period of the trial, colesevelam (tablets), as monotherapy or with a statin, was an effective treatment for pediatric patients with heterozygous familial hypercholesterolemia. At week 8, recipients of colesevelam 3.75 g/day had significant percentage reductions from baseline in mean LDL-C levels (primary endpoint) compared with placebo recipients. Significant beneficial treatment effects for colesevelam 3.75 g/day versus placebo were also reported for several other lipid/lipoprotein parameters at week 8 of the study. The reported treatment effects on lipid/lipoprotein parameters were maintained over a subsequent 18-week, open-label, noncomparative period, when all patients received colesevelam 3.75 g/day. Colesevelam 3.75 g/day was generally well tolerated for up to 26 weeks by pediatric patients with heterozygous familial hypercholesterolemia.

RCT Entities:   Population Interventions Outcomes