Cardiology in the Young

Original Articles

Treatment of familial hypercholesterolaemia in children and adolescents in the last three decades

Avishay Elisa1, Rong Zhoua2 and Evan A. Steina3

a1 Department of Medicine, Rabin Medical Center, Beilinson Campus, Petah-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel

a2 Medpace Inc., Cincinnati, Ohio, United States of America

a3 Metabolic & Atherosclerosis Research Center and Cholesterol Treatment Center, Cincinnati, Ohio, United States of America


Background: This study evaluated the effectiveness of long-term intensive lipid-lowering therapy in children and adolescents with familial hypercholesterolaemia. Methods: The charts of 89 children and adolescents with heterozygous familial hypercholesterolaemia among ∼1000 patients treated from 1974 to 2008 were reviewed. Familial hypercholesterolaemia was defined as low-density lipoprotein cholesterol level >90th percentile in individuals with a history of familial hypercholesterolaemia. Results: Of the 89 patients, 51% were male; the mean age at diagnosis was 8 ± 4 years, and the mean follow-up was 13 ± 8 years. Baseline and most recent low-density lipoprotein cholesterol levels (mg/dl) under treatment were 250 ± 50 and 142 ± 49, respectively, reduced 43% from baseline (p < 0.0001). At the most recent visit, 39 patients received statin monotherapy, mainly atorvastatin or rosuvastatin, and 50 (56%) patients received combination therapy, mainly vytorin or rosuvastain/ezetimibe, 15 patients were >30 years of age, and none developed symptomatic cardiovascular disease or needed revascularisation. Conclusions: Long-term statin-based therapy can reduce low-density lipoprotein cholesterol levels in most children and adolescents with heterozygous familial hypercholesterolaemia and decrease cardiovascular risk significantly.

(Received November 18 2012)

(Accepted March 31 2013)

(Online publication May 10 2013)


  • Familial hypercholesterolaemia;
  • heterozygous familial hypercholesterolaemia;
  • lipid-lowering therapy;
  • statin-based therapy


c1 Correspondence to: A. Elis, MD, Department of Medicine, Kfar Saba and Sackler School of Medicine, Meir Medical Center, Kfar Saba 44281, Israel. Tel: 972-9-7472185; Fax: 972-9-7460781; E-mail: