Ezetimib – Selective Inhibitor of Cholesterol and Its Application in the Therapy of Familial Hypercholesterolemia in Children and Adolescents

Authors: J. Hyánek ;  V. Martiníková ;  J. Dvořáková ;  L. Dubská ;  F. Pehal
Authors‘ workplace: Metabolická ambulance, Oddělení klinické biochemie, hematologie, imunologie a molekulární genetiky ;  Nemocnice Na Homolce, Praha přednosta prim. MUDr. M. Průcha, PhD.
Published in: Čes-slov Pediat 2007; 62 (11): 603-609.
Category: Original Papers


The search for new metabolically effective and harmless hypolipidemic drugs for the therapy of familial hypercholesterolemia proved to be successful in finding a selective inhibitor of cholesterol absorption, which was tested in our children and adolescents for the first time.

Practical testing of a new non-statin drug ezetimib – a selective inhibitor of intestinal absorption of cholesterol – for efficient therapy of the child familial hypercholesterolemia.

Twenty-five children with familial hypercholesterolemia demonstrated by molecular genetic methods were successfully treated with ezetimib at the dose of 5 or 10 mg/d as monotherapy or in combination with simvastatin or atorvastatin (2.5 to 20 mg/d). In all patients the therapeutic level of total cholesterol of 4.8 mmol/l was easily reached. In 15 children Ezetrol (5 to 10 mg/d) alone was sufficient for cholesterol levels to become normal. No unfavorable effects have been observed during the two years of therapy.

Ezetrol (Schering Plough-MSD) administered at the dose of 5 or 10 mg/d as monotherapy or in combination with simvastatin or atorvastatin very efficiently decreased the level of elevated cholesterol in the child and adolescent hypercholesterolemia to therapeutic level. Over the two years of administration neither untoward side effects have been observed, nor changes in laboratory markers of nutrition including vitamins, sex hormones or somatometric data of growth and body mass.

Key words:
familial hypercholesterolemia in children and adolescents, ezetimib, drug therapy, non-statin therapy

Neonatology Paediatrics General practitioner for children and adolescents
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