title: [Use of ezetimibe in the treatment of familial hypercholesterolemia in children and adolescents].
abstract: Heterozygous familial hypercholesterolemia (FH) is the most common inherited type of primary hyperlipidemia. Patients with familial hypercholesterolemia have an increased level of LDL cholesterol since childhood, and present early associated cardiovascular disease. Ezetimibe reduces LDL by blocking sterol absorption in enterocytes.
  
  
  OBJECTIVE
  to show our experience on the use of ezetimibe in children and adolescents with familial hypercholesterolemia, with short and medium term follow-up.
  
  
  METHODS
  Retrospective and longitudinal study. Patients who were receiving ezetimibe as monotherapy from 2003 to 2009 were included. The primary efficacy parameter was the effect of ezetimibe on the LDL after three months of treatment. Serum levels of aspartate aminotransferase, alanine aminotransferase and creatine kinase were monitored. Patients were asked if they experienced any side effect with the ezetimibe treatment. If the Patients did not achieve therapeutical goals with ezetimibe as monotherapy a statin was added. Outcome at medium term follow-up is analysed.
  
  
  RESULTS
  The study included a total of 32 patients. The mean age at the start of ezetimibe was 9.5 years (range: 2 to 15.5). The mean total time of Ezetimibe was 2.45 years (r: 0.4 - 5.9).The decrease in mean LDL levels was -25.7% ± 12.3 or 59.5 ± 34 mg% (P<.0001; 95% CI: 47.3-71.5, t test). There were no side effects with ezetimibe monotherapy. At the end of the study, 11 patients required added statins due to failing to achieve the treatment goal.
  
  
  CONCLUSIONS
  Ezetimibe is effective and safe for children and adolescents with FH in short and medium term follow-up.
authors.affiliation: Servicio de Nutrición, Hospital Nacional de Pediatría J. P. Garrahan, Buenos Aires, Argentina. maribearaujo@yahoo.com.ar
authors.forename: M B
authors.initials: MB
authors.lastname: Araujo
authors.forename: P M
authors.initials: PM
authors.lastname: Botto
authors.forename: C S
authors.initials: CS
authors.lastname: Mazza
mesh_terms: D000293:Adolescent; D000924:Anticholesteremic Agents / Q000627:therapeutic use*; D001384:Azetidines / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D000069438:Ezetimibe; D005260:Female; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy*; D008137:Longitudinal Studies; D008297:Male; D012189:Retrospective Studies
chemical_list: D000924:Anticholesteremic Agents; D001384:Azetidines; D000069438:Ezetimibe