title: Colesevelam hydrochloride: efficacy and safety in pediatric subjects with heterozygous familial hypercholesterolemia.
abstract: OBJECTIVE
  Evaluate the efficacy and safety of colesevelam hydrochloride in children with heterozygous familial hypercholesterolemia (heFH).
  
  
  METHODS
  This was a randomized, double-blind, 41-site study in 194 children aged 10 to 17 years (inclusive) with heFH (statin-naïve or on a stable statin regimen). After a 4-week stabilization period (period I), subjects were randomized 1:1:1 to placebo, colesevelam 1.875 g/d, or colesevelam 3.75 g/d for 8 weeks (period II). All then received open-label colesevelam 3.75 g/d for 18 weeks (period III), with follow-up 2 weeks later. The primary endpoint was percent change in low-density lipoprotein (LDL)-cholesterol from baseline to week 8. Secondary endpoints included percent change in other lipoprotein variables, including non-high-density lipoprotein (non-HDL)-cholesterol. Adverse events were also evaluated.
  
  
  RESULTS
  At week 8, a significant difference from baseline in LDL-cholesterol was reported with colesevelam 1.875 g/d (-6.3%; P = .031) and colesevelam 3.75 g/d (-12.5%; P < .001) compared with placebo. Significant treatment effects were also reported for total cholesterol (-7.4%), non-HDL-cholesterol (-10.9%), HDL-cholesterol (+6.1%), apolipoprotein A-I (+6.9%), and apolipoprotein B (-8.3%) and a nonsignificant effect for triglycerides (+5.1%) with colesevelam 3.75 g/d compared with placebo at week 8. These treatment effects were maintained during period III.
  
  
  CONCLUSIONS
  Colesevelam significantly lowered LDL-cholesterol levels in children with heFH.
authors.affiliation: Metabolic and Atherosclerosis Research Center, 4685 Forest Ave., Suite A, Cincinnati, OH 45212, USA. ESteinMRL@aol.com
authors.forename: Evan A
authors.initials: EA
authors.lastname: Stein
authors.forename: A David
authors.initials: AD
authors.lastname: Marais
authors.forename: Tamas
authors.initials: T
authors.lastname: Szamosi
authors.forename: Frederick J
authors.initials: FJ
authors.lastname: Raal
authors.forename: Daniel
authors.initials: D
authors.lastname: Schurr
authors.forename: Elaine M
authors.initials: EM
authors.lastname: Urbina
authors.forename: Paul N
authors.initials: PN
authors.lastname: Hopkins
authors.forename: Sulekha
authors.initials: S
authors.lastname: Karki
authors.forename: Jianbo
authors.initials: J
authors.lastname: Xu
authors.forename: Soamnauth
authors.initials: S
authors.lastname: Misir
authors.forename: Michael
authors.initials: M
authors.lastname: Melino
mesh_terms: D000293:Adolescent; D000499:Allylamine / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D002648:Child; D008078:Cholesterol, LDL / Q000097:blood / Q000187:drug effects; D000069472:Colesevelam Hydrochloride; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004359:Drug Therapy, Combination; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000008:administration & dosage; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy*; D008297:Male
chemical_list: D000924:Anticholesteremic Agents; D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D000499:Allylamine; D000069472:Colesevelam Hydrochloride