title: The efficacy of colesevelam HCl in the treatment of heterozygous familial hypercholesterolemia in pediatric and adult patients.
abstract: BACKGROUND
  Familial hypercholesterolemia (FH) is a common autosomal co-dominant genetic disorder that results in severely increased levels of LDL-C. Patients with FH are at an increased risk for premature coronary artery disease. Expert panels therefore recommend initiation of lipid-lowering therapy in childhood to reduce the very high lifetime risk of coronary artery disease. The bile acid sequestrant colesevelam is indicated to reduce elevated LDL-C levels in adults with primary hyperlipidemia and in boys and postmenarchal girls (aged 10-17 years) with heterozygous FH.
  
  
  OBJECTIVE
  The purpose of this article was to review currently available data on the use of colesevelam in the treatment of heterozygous FH.
  
  
  METHODS
  PubMed and Google Scholar were searched to identify clinical trials evaluating colesevelam in patients with heterozygous FH.
  
  
  RESULTS
  The search returned 2 results (both multicenter, multinational studies): 1 study conducted in adults and the other in pediatric patients. In the study in adults with refractory FH, the addition of colesevelam to a maximally tolerated regimen of a statin plus ezetimibe provided a significantly greater reduction from baseline in LDL-C levels compared with placebo. Significantly greater reductions from baseline in LDL-C were also seen in pediatric patients with heterozygous FH receiving colesevelam (alone or in combination with statins) compared with placebo. Colesevelam was generally well tolerated in studies in patients with FH; consistent with other colesevelam studies, gastrointestinal disorders were the most common drug-related adverse events, but these events rarely led to study withdrawal.
  
  
  CONCLUSIONS
  Currently available data demonstrate that colesevelam, alone or in combination therapy, is efficacious and well tolerated in the treatment of heterozygous FH in adults and pediatric patients, supporting its use as a treatment option in both of these patient populations.
authors.affiliation: University of Chicago, Pritzker School of Medicine, Chicago, IL 60611, USA. mdavidso@medicine.bsd.uchicago.edu
authors.forename: Michael
authors.initials: M
authors.lastname: Davidson
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D055815:Young Adult; D000499:Allylamine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use; D000924:Anticholesteremic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D001384:Azetidines / Q000627:therapeutic use; D008078:Cholesterol, LDL / Q000097:blood; D000069472:Colesevelam Hydrochloride; D003324:Coronary Artery Disease / Q000150:complications / Q000188:drug therapy; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D000069438:Ezetimibe; D005767:Gastrointestinal Diseases / Q000139:chemically induced; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000627:therapeutic use; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy
chemical_list: D000499:Allylamine; D000924:Anticholesteremic Agents; D001384:Azetidines; D008078:Cholesterol, LDL; D000069472:Colesevelam Hydrochloride; D000069438:Ezetimibe; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors
keywords: LDL-C; adolescents; children; colesevelam; familial hypercholesterolemia