Journal Article

The Efficacy of Colesevelam HCl in the Treatment of Heterozygous Familial Hypercholesterolemia in Pediatric and Adult Patients

Davidson, Michael

2013

Overview

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. The purpose of this article was to review currently available data on the use of colesevelam in the treatment of heterozygous FH. PubMed and Google Scholar were searched to identify clinical trials evaluating colesevelam in patients with heterozygous FH. 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. 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.

• Publisher: EM Inc USA,Elsevier,Elsevier Limited
• Subject: Acids / Adolescent / adolescents / Adult / Aged / Allylamine - administration & dosage / Allylamine - adverse effects / Allylamine - analogs & derivatives / Allylamine - therapeutic use / Anticholesteremic Agents - administration & dosage / Anticholesteremic Agents - adverse effects / Anticholesteremic Agents - therapeutic use / Apolipoproteins / Azetidines - therapeutic use / Bile / Biological and medical sciences / Cardiovascular disease / children / Cholesterol / Cholesterol, LDL - blood / Clinical trials / colesevelam / Colesevelam Hydrochloride / Coronary Artery Disease - complications / Coronary Artery Disease - drug therapy / Disorders of blood lipids. Hyperlipoproteinemia / Double-Blind Method / Drug Administration Schedule / Drug Therapy, Combination / Enzymes / Ezetimibe / familial hypercholesterolemia / Female / Gastrointestinal Diseases - chemically induced / Humans / Hydroxymethylglutaryl-CoA Reductase Inhibitors - therapeutic use / Hyperlipoproteinemia Type II - drug therapy / Internal Medicine / LDL-C / Lipids / Lipoproteins / Male / Medical Education / Medical sciences / Metabolic diseases / Middle Aged / Pharmacology. Drug treatments / Randomized Controlled Trials as Topic / Rodents / Studies / Triglycerides / Young Adult