Personalized treatment and prophylactic strategies in pediatric patients from the Ukrainian familial hypercholesterolemia registry: a comprehensive study
DOI:
https://doi.org/10.15574/SP.2024.8(144).5564Keywords:
children, familial hypercholesterolemia, personalized treatment, lipid profile, cardiovascular risk, statins, dietary intervention, pediatric dyslipidemiaAbstract
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.
References
Corder K, van Sluijs EMF, Wright A, Whincup P, Wareham NJ, Ekelund U. (2009). Is it possible to assess free-living physical activity and energy expenditure in young people by self-report? The American Journal of Clinical Nutrition. 89(3): 862-870. https://doi.org/10.3945/ajcn.2008.26739; PMid:19144732
De Ferranti SD et al. (2019). Cardiovascular risk reduction in high-risk pediatric patients: A scientific statement from the American heart association. Circulation, 139(13). https://doi.org/10.1161/CIR.0000000000000618
European Association for Cardiovascular Prevention & Rehabilitation, Reiner Z, Catapano AL, De Backer G, Graham I et al. (2011). ESC/EAS Guidelines for the management of dyslipidaemias: The Task Force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). European Heart Journal. 32(14): 1769-1818. https://doi.org/10.1093/eurheartj/ehr158; PMid:21712404
Expert Panel on Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents, National Heart, Lung, and Blood Institute (2011). Expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents: summary report. Pediatrics. 128; Suppl 5: S213-S256. https://doi.org/10.1542/peds.2009-2107C; PMid:22084329 PMCid:PMC4536582
Harrell JS, Mcmurray RG, Baggett CD, Pennell ML, Pearce PF, Bangdiwala SI. (2005). Energy costs of physical activities in children and adolescents. Medicine and Science in Sports and Exercise. 37(2): 329-336. https://doi.org/10.1249/01.MSS.0000153115.33762.3F; PMid:15692331
Kavey RE. (2024) Dyslipidemia in children and adolescents: Definition, screening, and diagnosis. UpToDate [Internet]. Waltham, MA: UpToDate; URL: https://www.uptodate.com/contents/dyslipidemia-inchildren-and-adolescents-definition-screening-and-diagnosis.
Kulchytska Y-EB. (2023). Evaluation of clinical and diagnostic significance of dyslipidemia-based prognostic model for cardiovascular lesions in pediatric patients. Dissertation for the scientific degree of Candidate of Medical Sciences (Doctor of Philosophy), Shupyk National Healthcare University of Ukraine. Shupyk NHUU Repository. URL: https://www.nuozu.edu.ua/zagruzka3/Dr_Kulhicka.pdf.
Kulchytska Y-EВ, Marushko TV, Kurilina TV. (2023). Health-related quality of life and physical activity in Ukrainian pediatric patients with heterozygous familial hypercholesterolemia. Modern Pediatrics. Ukraine. 2(130): 24-31. https://doi.org/10.15574/SP.2023.130.24
LibGuides. (2013). SPSS tutorials: Pearson Correlation. URL: https://libguides.library.kent.edu/SPSS/PearsonCorr.
Marushko TV, Kurilina TV, Kulchytska Y-EB. (2022). Lipid profile peculiarities and matrix Gla protein concentration in Ukrainian pediatric patients with heterozygous familial hypercholesterolemia. Modern Pediatrics. Ukraine. 8(128): 12-20. https://doi.org/10.15574/SP.2022.128.12
Marushko T, Kurilina T, Kulchytska Y-E. (2023). Impact of the Cardiovascular Health Integrated Lifestyle Diet on nutritional profile and dietary compliance in Ukrainian pediatric patients with heterozygous familial hypercholesterolemia. Child's health. 17(8): 374-381. https://doi.org/10.22141/2224-0551.17.8.2022.1543
Marushko T, Kurilina T, Kulchytska Y-E. (2024). Vascular Microcalcification: Diagnostic Approach, Statistical Modeling, and the Need for Comprehensive Management of Children from the Ukrainian Familial Hypercholesterolemia Registry. Family medicine. European practices. (4): 108-113. https://doi.org/10.30841/2786-720X.4.2024.320822
McCrindle BW. (2007). Summary of the American heart association's scientific statement on drug therapy of high-risk lipid abnormalities in children and adolescents. Arteriosclerosis, Thrombosis, and Vascular Biology. 27(5): 982-985. https://doi.org/10.1161/ATVBAHA.107.143644; PMid:17442893
Mulligan AA, Luben RN, Bhaniani A, Parry-Smith DJ, O'Connor L, Khawaja AP et al. (2014). A new tool for converting food frequency questionnaire data into nutrient and food group values: FETA research methods and availability. BMJ Open. 4(3): e004503. https://doi.org/10.1136/bmjopen-2013-004503; PMid:24674997 PMCid:PMC3975761
Pourebrahim R et al. (2006). Household cardiovascular screening of high-risk families: a school-based study. European Journal of Cardiovascular Prevention and Rehabilitation: Official Journal of the European Society of Cardiology, Working Groups on Epidemiology & Prevention and Cardiac Rehabilitation and Exercise Physiology. 13(2): 229-235. https://doi.org/10.1097/01.hjr.0000214605.53372.62; PMid:16575277
Ravens-Sieberer U, Bullinger M. (1998). Assessing health-related quality of life in chronically ill children with the German KINDL: first psychometric and content analytical results. Quality of Life Research: An International Journal of Quality of Life Aspects of Treatment, Care and Rehabilitation. 7(5): 399-407. https://doi.org/10.1023/A:1008853819715; PMid:9691720
Sesso HD, Paffenbarger RS Jr, Lee I-M. (2000). Physical activity and coronary heart disease in men: The Harvard alumni health study. Circulation. 102(9): 975-980. https://doi.org/10.1161/01.CIR.102.9.975; PMid:10961960
Vallejo-Vaz AJ et al. (2018). Overview of the current status of familial hypercholesterolaemia care in over 60 countries - The EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Atherosclerosis. 277: 234-255. https://doi.org/10.1016/j.atherosclerosis.2018.08.051; PMid:30270054
Yeste D, Chacón P, Clemente M, Albisu MA, Gussinyé M, Carrascosa A. (2009). Ezetimibe as monotherapy in the treatment of hypercholesterolemia in children and adolescents. Journal of Pediatric Endocrinology & Metabolism. 22(6). https://doi.org/10.1515/JPEM.2009.22.6.487
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