Colesevelam hydrochloride: efficacy and safety in pediatric subjects with heterozygous familial hypercholesterolemia.

摘要: Objective Evaluate the efficacy and safety of colesevelam hydrochloride in children with heterozygous familial hypercholesterolemia (heFH). Study design This was a randomized, double-blind, 41-site study 194 aged 10 to 17 years (inclusive) heFH (statin-naive or on stable statin regimen). After 4-week stabilization period (period I), subjects were randomized 1:1:1 placebo, 1.875 g/d, 3.75 g/d for 8 weeks II). All then received open-label 18 III), follow-up 2 later. The primary endpoint percent change low-density lipoprotein (LDL)-cholesterol from baseline week 8. Secondary endpoints included other variables, including non-high-density (non-HDL)-cholesterol. Adverse events also evaluated. Results At 8, significant difference LDL-cholesterol reported 1.875 g/d (−6.3%; P = .031) (−12.5%; Conclusions Colesevelam significantly lowered levels heFH.