Personalized treatment and prophylactic strategies in pediatric patients from the Ukrainian familial hypercholesterolemia registry: a comprehensive study

Familial hypercholesterolemia (FH) is a genetic disorder characterized by elevated low-density lipoprotein (LDL) levels, increasing the risk of premature cardiovascular disease. Personalized treatment strategies tailored to pediatric FH patients offer promising solutions for effective management. This study aimed to develop and evaluate a personalized treatment and prophylactic program focused on managing lipid levels and reducing cardiovascular risks in children with FH. Materials and methods. The study included 15 children aged 5-18 years diagnosed with FH. A comprehensive evaluation was conducted, assessing anthropometric data, dietary profiles, and compliance, quality of life, and physical activity energy expenditure (PAEE). An extended lipid profile analysis included ApoA1, ApoB, lipoprotein(a), and dp-ucMGP levels. Cardiovascular risks were evaluated using instrumental assessments. Patients were divided into age groups, and personalized interventions were implemented - medical nutrition therapy, pharmacotherapy (statins, Omega-3 fatty acids, ezetimibe), and lifestyle modifications. Statistical analysis was performed using SAS® OnDemand for Academics. Results. A stepwise diagnostic and therapeutic algorithm for managing children with pediatric dyslipidemia was developed. Logistic regression analysis revealed statistical significance in personalized interventions, with a positive β coefficient of 1.34. The odds ratio for statins was 3.82, indicating that their inclusion increased the likelihood of achieving target LDL-C levels by nearly fourfold. PAEE showed a trend toward influencing LDL-C achievement. Conclusions. Personalized treatment strategies, combining dietary correction and targeted pharmacotherapy, effectively improved lipid profiles and reduced cardiovascular risks in children with FH. Significant external factors impacting LDL-C targets were identified. This study highlights the importance of individualized approaches in pediatric FH care and underscores the need for further research. This research was carried out in accordance with the principles of the Declaration of Helsinki. The study protocol was approved by the Local Ethical Committee. The informed consent was obtained from all participants. No conflict of interest was declared by the authors.