A promising agent for lipid profile correction in children with familial hypercholesterolemia

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.