A promising agent for lipid profile correction in children with familial hypercholesterolemia
https://doi.org/10.21518/ms2025-058
Abstract
Introduction. Heterozygous familial hypercholesterolemia (heFH) is a disorder with a high risk of early cardiovascular events and a fatal risk of myocardial infarction at the age of 20–39 years.
Aim. To assess the efficacy and safety of the use of ezetimibe in real-world settings in children and adolescents with heFH, and to compare the efficacy of ezetimibe monotherapy and combination therapy with statins in this population
Materials and methods. A retrospective descriptive analysis study was conducted from August 2021 to December 2024. The study assessed the efficacy of ezetimibe monotherapy and ezetimibe and atorvastatin combination therapy in 130 children with dyslipidaemia (average age 13.2 ± 3.1 years). Prior to treatment initiation all subjects followed a lipid-lowering diet, after which their baseline lipid profile was determined.
Results. The results showed that 3-month ezetimibe monotherapy led to a decrease in the levels of total cholesterol (TC), low-density lipoprotein cholesterol (LDL-C), and non-high-density lipoprotein cholesterol (non-HDL-C), which was statistically significant. The average LDL-C reduction was 1.6 ± 0.6 mmol/L (21.3%), and LDL-C targets (<4 mmol/L for children <10 years and <3.5 mmol/L for children ≥10 years) were achieved in 70% of patients. In patients with an inadequate response to ezetimibe monotherapy, atorvastatin or combination therapy were prescribed, which demonstrated a 46.9% and 53.1% decrease in LDL-C levels from baseline, respectively, which were statistically significant.
Discussion. Ezetimibe monotherapy resulted in a decrease in TC, LDL-C and non-HDL-C levels. These results were consistent with the results from the previous studies and showed the efficacy of ezetimibe as a first-line drug to treat hypercholesterolemia in children. However, the monotherapy was less-than-adequate in 30% of patients.
Conclusion. The results of the study have confirmed the efficacy and safety of ezetimibe monotherapy in children with dyslipidaemia. It was difficult to achieve target values in patients with high baseline LDL-C levels using monotherapy, which justifies the necessity of combination therapy.
About the Authors
I. I. Pshenichnikova
Russian Medical Academy of Continuous Professional Education; Bashlyaeva Children’s City Clinical Hospital; Research Institute of Healthcare Organization and Medical Management
Russian Federation
Russian Federation
Irina I. Pshenichnikova - Cand. Sci. (Med.), Associate Professor of the Department of Pediatrics named after Academician G.N. Speransky, Russian Medical Academy of Continuing Professional Education; Specialist of the Organizational and Methodical Department of Paediatrics, Research Institute of Healthcare Organization and Medical Management; Bashlyaeva Children’s City CH, Moscow Healthcare Department; Paediatrician, Bashlyaeva Children’s City CH.
2/1, Bldg. 1, Barrikadnaya St., Moscow, 125993; 9, Sharikopodshipnikovskaya St., Moscow, 115088; 28, Geroyev Panfilovtsev St., Moscow, 125373
I. N. Zakharova
Russian Medical Academy of Continuous Professional Education
Russian Federation
Russian Federation
Irina N. Zakharova - Dr. Sci. (Med.), Professor, Head of the Department of Pediatrics named after G.N. Speransky.
2/1, Bldg. 1, Barrikadnaya St., Moscow, 125993
I. N. Trunina
Bashlyaeva Children’s City Clinical Hospital; Pirogov Russian National Research Medical University
Russian Federation
Russian Federation
Inna I. Trunina - Dr. Sci. (Med.), Professor, Head of the Cardiology Department, Bashlyaeva Children’s City Clinical Hospital; Professor of Department of Hospital Paediatrics No 1, Faculty of Paediatrics.
28, Geroyev Panfilovtsev St., Moscow, 125373; 2, Taldomskaya St., Moscow, 125412
V. V. Pupykinа
Russian Medical Academy of Continuous Professional Education
Russian Federation
Russian Federation
Viktoria V. Pupykina - Postgraduate Student of the Department of Pediatrics named after Academician G.N. Speransky.
2/1, Bldg. 1, Barrikadnaya St., Moscow, 125993
M. V. Ezhov
National Medical Research Centre of Cardiology named after Academician E.I. Chazov
Russian Federation
Russian Federation
Marat V. Ezhov - Dr. Sci. (Med.), Professor, Chief Researcher, Laboratory of Lipid Metabolism Disorders, Myasnikov Research Institute of Clinical Cardiology.
15a, Academician Chazov St., Moscow, 121552
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Review
For citations:
Pshenichnikova II, Zakharova IN, Trunina IN, Pupykinа VV, Ezhov MV. A promising agent for lipid profile correction in children with familial hypercholesterolemia. Meditsinskiy sovet = Medical Council. 2025;(1):187-194. (In Russ.) https://doi.org/10.21518/ms2025-058
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