title: A 10-year experience using combined lipid-lowering pharmacotherapy in children and adolescents.
abstract: BACKGROUND
  Current pediatric guidelines for heterozygous familial hypercholesterolemia (HeFH) propose pharmacotherapy (PT) with statins from age 8 to 10 years; however, schemes with absorption inhibitors combined with statins, could be started earlier. The aim of the study was to show the 10-year results of a combined treatment protocol.
  
  
  METHODS
  Prospective, descriptive and analytical study. Pediatric patients (n=70; mean age at PT initiation 9.3 years [range, 2-17.5]) with HeFH who required PT between 2005 and 2015 were included. All patients ≥10 years, with LDL >190 mg/dL or >160 mg/dL with one cardiovascular risk factor (CVRF) or >130 mg/dL with two or more CVRF; and those patients 5-10 years and with LDL-C >240 mg/dL or a family history of a cardiovascular event before 40 years, were medicated. After a period on a lipid-lowering diet (LLD), all patients were started on ezetimibe. Patients who did not achieve the treatment goal were given statins. The variables were: age, age at PT initiation, duration of PT, initial LDL-C, mean LDL-C during ezetimibe monodrug therapy, mean LDL-C during combined PT, and percentage of LDL decrease.
  
  
  RESULTS
  LDL-C levels were: Baseline: 235 mg/dL±55; after 3 months on ezetimibe: 167 mg/dL±47 (decrease: -27.62%). In 18 patients who did not reach the treatment goal atorvastatin was added and their LDL-C decreased -41.5% (p: 0.02). Overall, mean final LDL-C was 155 mg/dL±30.4 (range, 98-257) and treatment goals were reached in 74% of the patients. No severe side effects were reported.
  
  
  CONCLUSIONS
  Combined and sequential treatment starting at early ages was shown to be safe and effective over this follow-up period.
authors.forename: María Beatriz
authors.initials: MB
authors.lastname: Araujo
authors.forename: María Sol
authors.initials: MS
authors.lastname: Pacce
mesh_terms: D000293:Adolescent; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001118:Argentina / Q000453:epidemiology; D002318:Cardiovascular Diseases / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control*; D002648:Child; D008078:Cholesterol, LDL / Q000097:blood; D003131:Combined Modality Therapy / Q000009:adverse effects; D018752:Diet, Fat-Restricted / Q000009:adverse effects; D016903:Drug Monitoring; D004359:Drug Therapy, Combination / Q000009:adverse effects; D000069438:Ezetimibe / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006776:Hospitals, Pediatric; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000009:adverse effects / Q000627:therapeutic use*; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000178:diet therapy / Q000188:drug therapy* / Q000503:physiopathology; D008077:Lipoproteins, LDL / Q000097:blood; D008297:Male; D010044:Outpatient Clinics, Hospital; D017410:Practice Guidelines as Topic; D011446:Prospective Studies; D012307:Risk Factors; D012720:Severity of Illness Index
chemical_list: D000924:Anticholesteremic Agents; D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D008077:Lipoproteins, LDL; D000069438:Ezetimibe