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Journal of Pediatric Endocrinology and Metabolism

Editor-in-Chief: Kiess, Wieland

Ed. by Bereket, Abdullah / Darendeliler, Feyza / Dattani, Mehul / Gustafsson, Jan / Luo, Fei Hong / Toppari, Jorma / Turan, Serap Demircioglu

IMPACT FACTOR 2018: 1.239

CiteScore 2018: 1.22

SCImago Journal Rank (SJR) 2018: 0.507
Source Normalized Impact per Paper (SNIP) 2018: 0.562

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Volume 29, Issue 11


A 10-year experience using combined lipid-lowering pharmacotherapy in children and adolescents

María Beatriz Araujo / María Sol Pacce
Published Online: 2016-10-08 | DOI: https://doi.org/10.1515/jpem-2016-0117



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.


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.


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.


Combined and sequential treatment starting at early ages was shown to be safe and effective over this follow-up period.

Keywords: ezetimibe; heterozygous familial hypercholestrolemia; pediatrics; pharmacotherapy; statins


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About the article

Received: 2016-04-01

Accepted: 2016-09-05

Published Online: 2016-10-08

Published in Print: 2016-11-01

Author contributions: All the authors have accepted responsibility for the entire content of this submitted manuscript and approved submission.

Research funding: None declared.

Employment or leadership: None declared.

Honorarium: None declared.

Competing interests: The funding organization(s) played no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication.

Citation Information: Journal of Pediatric Endocrinology and Metabolism, Volume 29, Issue 11, Pages 1285–1291, ISSN (Online) 2191-0251, ISSN (Print) 0334-018X, DOI: https://doi.org/10.1515/jpem-2016-0117.

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