Ezetimibe in the treatment of dyslipidemia in children and adolescents with types 1 and 2 diabetes mellitus: efficacy and safety

Pshenichnikova I.I., Zakharova I.N., Borzakova S.N., Pupykina V.V., Osmanov I.M.

1) Russian Medical Academy of Continuous Professional Education, Moscow, Russia; 2) Z.A. Bashlyaeva Children’s City Clinical Hospital, Moscow, Russia; 3) Research Institute for Healthcare Organization and Medical Management, Moscow, Russia

Objective: Systematization of the data on the efficacy and safety of ezetimibe in patients aged 6 to 18 years with type 1 (DM1) and type 2 (DM2) diabetes mellitus, and to compare the findings with current clinical guidelines.
Methods: A narrative literature review using the key words «ezetimibe,» «children,» «adolescents,» «diabetes,» «dyslipidemia,» and «low-density lipoprotein» in PubMed, Embase, the Cochrane Library, and ClinicalTrials.gov (January 2000 – June 2025) was conducted.
Results: A total of 34 publications were analyzed, including 6 randomized controlled trials (RCTs), 12 observational studies, 5 systematic reviews/meta-analyses, 4 clinical guidelines/consensus papers, 6 reviews and reference articles, and 1 registry document. A single pediatric RCT (n=138, 6–10 years; predominantly heterozygous familial hypercholesterolemia (heFH)) showed a 27% reduction in low-density lipoprotein (LDL) cholesterol with ezetimibe monotherapy, with a safety profile comparable to placebo. In a small crossover study in adolescents with DM1, ezetimibe monotherapy provided a greater reduction in LDL cholesterol compared to simvastatin (≈13 pp); the combination of a statin and ezetimibe was not evaluated in this study. There are no pediatric RCTs in DM2. A network meta-analysis (n=1649; pediatric cohorts with heFH) found that adding ezetimibe to a statin reduced LDL cholesterol by an additional ~16% without increasing the risk of adverse effects. These results are extrapolated to adolescents with diabetes using an individual risk assessment.
Conclusion: Long-term studies of the efficacy and safety of ezetimibe in children with DM2 are required.

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About the Authors

Irina I. Pshenichnikova, Cand. Sci. (Med.), Associate Professor, Speransky Department of Pediatrics, Russian Medical Academy of Continuous Professional Education; Pediatric Cardiologist, Z.A. Bashlyaeva Children’s City Clinical Hospital; Specialist, Research Institute for Healthcare Organization and Medical Management, Moscow, Russia; PshenichnikovaII@rmapo.ru, ORCID: https://orcid.org/0000-0002-0058-3803 (corresponding author)
I.N. Zakharova, Dr. Sci. (Med.), Professor, Head of the Speransky Department of Pediatrics, Russian Medical Academy of Continuous Professional Education, Moscow, Russia, Honored Doctor of the Russian Federation, zakharova-rmapo@yandex.ru, ORCID: https://orcid.org/0000-0003-4200-4598
S.N. Borzakova, Cand. Sci. (Med.), Associate Professor, Speransky Department of Pediatrics, Russian Medical Academy of Continuous Professional Education; Gastroenterologist, Z.A. Bashlyaeva Children’s City Clinical Hospital; Leading Specialist, Research Institute for Healthcare Organization and Medical Management, Moscow, Russia; borzakovasn@zdrav.mos.ru, ORCID: https://orcid.org/0000-0001-5544-204X
V.V. Pupykina, Teaching Assistant, Speransky Department of Pediatrics, Russian Medical Academy of Continuous Professional Education, Moscow, Russia; vika-pupykina@mail.ru, ORCID: https://orcid.org/0000-0003-2181-8138
I.M. Osmanov, Dr. Sci. (Med.), Professor, Chief Physician, Z.A. Bashlyaeva Children’s City Clinical Hospital, Moscow, Russia; OsmanovIM@zdrav.mos.ru,
ORCID: https://orcid.org/0000-0003-3181-9601

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