STATIN TREATMENT AND POTENCY ROLE IN CHILDREN WITH FAMILIAL HYPERCHOLESTEROLEMIA: A REVIEW OF EFFICACY AND SAFETY
Main Article Content
Keywords
Hyperlipoproteinemia Type II, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Children
Abstract
Familial hypercholesterolemia (FH)is a genetic disorder that causes elevated levels of low-density lipoprotein cholesterol (LDL-C), increasing the risk of premature cardiovascular disease (CVD) at an early age. FH is classified into two forms: heterozygous FH (HeFH) and homozygous FH (HoFH). The identification of FH in children is crucial, as untreated patients have a higher risk of developing CVD than the general population. Statins are the first-line agents for FH treatment in children and aim to lower LDL-C levels and reduce the risk of CVD, especially in young adults. This review aims to provide a rationale for statin therapy in the management of pediatric FH and discusses the potential benefits and risks associated with statin efficacy. The article also discusses the overview of statins, statin potency, and the potential risks associated with statin therapy.
References
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https://doi.org/10.5772/intechopen.91367
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2. Ruel, I., Brisson, D., Aljenedil, S., Awan, Z., Baass, A., Bélanger, A., Bergeron, J., Bewick, D., Brophy, J. M., Brunham, L. R., Couture, P., Dufour, R., Francis, G. A., Frohlich, J., Gagné, C., Gaudet, D., Grégoire, J. C., Gupta, M., Hegele, R. A., Mancini, G. B. J., … Genest, J. (2018). Simplified Canadian Definition for Familial Hypercholesterolemia. The Canadian journal of cardiology, 34(9), 1210–1214. https://doi.org/10.1016/j.cjca.2018.05.015
3. Gidding, S. S., Champagne, M. A., de Ferranti, S. D., Defesche, J., Ito, M. K., Knowles, J. W., McCrindle, B., Raal, F., Rader, D., Santos, R. D., Lopes-Virella, M., Watts, G. F., Wierzbicki, A. S., & American Heart Association Atherosclerosis, Hypertension, and Obesity in Young Committee of Council on Cardiovascular Disease in Young, Council on Cardiovascular and Stroke Nursing, Council on Functional Genomics and Translational Biology, and Council on Lifestyle and Cardiometabolic Health (2015). The Agenda for Familial Hypercholesterolemia: A Scientific Statement from the American Heart Association. Circulation, 132(22), 2167–2192. https://doi.org/10.1161/CIR.0000000000000297
4. 19. Capra ME, Pederiva C, Banderali G, Biasucci G. Prevention starts from the crib: the paediatric point of view on detection of families at high cardiovascular risk. Ital J Pediatr. 2021;47:51. doi: 10.1186/s13052-021-00985-x. [PMC free article] [PubMed] [CrossRef] [Google Scholar]
5. Vallejo-Vaz, A. J., Kondapally Seshasai, S. R., Cole, D., Hovingh, G. K., Kastelein, J. J., Mata, P., Raal, F. J., Santos, R. D., Soran, H., Watts, G. F., Abifadel, M., Aguilar-Salinas, C. A., Akram, A., Alnouri, F., Alonso, R., Al-Rasadi, K., Banach, M., Bogsrud, M. P., Bourbon, M., Bruckert, E., … Ray, K. K. (2015). Familial hypercholesterolaemia: A global call to arms. Atherosclerosis, 243(1), 257–259. https://doi.org/10.1016/j.atherosclerosis.2015.09.021
6. Ann Marie Navar, Lawrence J. Fine, Walter T. Ambrosius, Arleen Brown, Pamela S. Douglas, Karen Johnson, Amit V. Khera, Donald Lloyd-Jones, Erin D. Michos, Mahasin Mujahid, Daniel Muñoz, Khurram Nasir, Nicole Redmond, Paul M Ridker, Jennifer Robinson, David Schopfer, Deborah F. Tate, Cora E. Lewis, Earlier treatment in adults with high lifetime risk of cardiovascular diseases: What prevention trials are feasible and could change clinical practice? Report of a National Heart, Lung, and Blood Institute (NHLBI) Workshop, American Journal of Preventive Cardiology, Volume 12, 2022,7.- Versmissen, J., Oosterveer, D. M., Yazdanpanah, M., Defesche, J. C., Basart, D. C., Liem, A. H., Heeringa, J., Witteman, J. C., Lansberg, P. J., Kastelein, J. J., & Sijbrands, E. J. (2008). Efficacy of statins in familial hypercholesterolaemia: a long term cohort study. BMJ (Clinical research ed.), 337, a2423.
https://doi.org/10.1136/bmj.a2423
7. 8.- Civeira, F., Arca, M., Cenarro, A., & Hegele, R. A. (2022). A mechanism-based operational definition and classification of hypercholesterolemia. Journal of clinical lipidology, 16(6), 813–821. https://doi.org/10.1016/j.jacl.2022.09.006
8. Endo A, Tsujita Y, Kuroda M, Tanzawa K. Inhibition of cholesterol synthesis in vitro and in vivo by ML-236A and ML-236B, competitive inhibitors of 3-hydroxy-3-methylglutaryl-coenzyme A reductase. Eur J Biochem.1977; 77:31–36.
9. Goldberg, A. C., Hopkins, P. N., Toth, P. P., Ballantyne, C. M., Rader, D. J., Robinson, J. G., Daniels, S. R., Gidding, S. S., de Ferranti, S. D., Ito, M. K., McGowan, M. P., Moriarty, P. M., Cromwell, W. C., Ross, J. L., Ziajka, P. E., & National Lipid Association Expert Panel on Familial Hypercholesterolemia (2011). Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. Journal of clinical lipidology, 5(3 Suppl), S1–S8. https://doi.org/10.1016/j.jacl.2011.04.003
10. McKenney J. M. (2003). Pharmacologic characteristics of statins. Clinical cardiology, 26(4 Suppl 3), III32–III38. https://doi.org/10.1002/clc.4960261507
11. Liu, A., Wu, Q., Guo, J., Ares, I., Rodríguez, J. L., Martínez-Larrañaga, M. R., Yuan, Z., Anadón, A., Wang, X., & Martínez, M. A. (2019). Statins: Adverse reactions, oxidative stress and metabolic interactions. Pharmacology & therapeutics, 195, 54–84.
12. Oesterle, A., Laufs, U., & Liao, J. K. (2017). Pleiotropic Effects of Statins on the Cardiovascular System. Circulation research, 120(1), 229–243.
https://doi.org/10.1161/CIRCRESAHA.116.308537
13. Chou R, Cantor A, Dana T, et al. Statin Use for the Primary Prevention of Cardiovascular Disease in Adults: A Systematic Review for the U.S. Preventive Services Task Force [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2022 Aug. (Evidence Synthesis, No. 219.) Table 1, Statin Dosing and ACC/AHA Classification of Intensity. Available from: https://www.ncbi.nlm.nih.gov/books/NBK583664/table/ch1.tab1/
14. Iyen B, Akyea RK, Weng S, Kai J, Qureshi N. Statin treatment and LDL-cholesterol treatment goal attainment among individuals with familial hypercholesterolaemia in primary care. Open Heart. 2021 Oct;8(2):e001817. doi: 10.1136/openhrt-2021-001817. PMID: 34702779; PMCID: PMC8549660.
15. Zhang, Q., Dong, J., Yu, Z. (2020). Pleiotropic use of Statins as non-lipid-lowering drugs. International Journal of Biological Sciences, 16(14), 2704-2711.
https://doi.org/10.7150/ijbs.42965.
16. Almeida, S. O., & Budoff, M. (2019). Effect of statins on atherosclerotic plaque. Trends in cardiovascular medicine, 29(8), 451–455. https://doi.org/10.1016/j.tcm.2019.01.001
17. Grundy S, Stone N, Bailey A, et al.
2018AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol. J Am Coll Cardiol. 2019 Jun, 73 (24) e285–e350.https://doi.org/10.1016/j.jacc.2018.11.003
18. Hussain, A., & Ballantyne, C. M. (2021). New Approaches for the Prevention and Treatment of Cardiovascular Disease: Focus on Lipoproteins and Inflammation. Annual review of medicine, 72, 431–446. https://doi.org/10.1146/annurev-med-100119-013612
19. Sunil, B., & Ashraf, A. P. (2020). Statin Therapy in Children. En A. Abukabda, M. Suciu, & M. Andor (Eds.), Cardiovascular Risk Factors in Pathology. IntechOpen.
https://doi.org/10.5772/intechopen.91367
20. Martin PD, Warwick MJ, Dane AL, Hill SJ, Giles PB, Phillips PJ, et al. Metabolism, excretion, and pharmacokinetics of rosuvastatin in healthy adult male volunteers. Clinical Therapeutics. 2003;25(11):2822-2835
21. Van der Graaf, A., Nierman, M. C., Firth, J. C., Wolmarans, K. H., Marais, A. D., & de Groot, E. (2006). Efficacy and safety of fluvastatin in children and adolescents with heterozygous familial hypercholesterolaemia. Acta paediatrica (Oslo, Norway : 1992), 95(11), 1461–1466. https://doi.org/10.1080/08035250600702602
22. Stein EA, Illingworth DR, Kwiterovich PO, et al. Efficacy and safety of lovastatin in adolescent males with heterozygous familial hypercholesterolemia: a randomized controlled trial. JAMA. 1999;281:137–143. [PubMed] [Google Scholar]
23. Knipscheer, H. C., Boelen, C. C., Kastelein, J. J., van Diermen, D. E., Groenemeijer, B. E., van den Ende, A., Büller, H. R., & Bakker, H. D. (1996). Short-term efficacy and safety of pravastatin in 72 children with familial hypercholesterolemia. Pediatric research, 39(5), 867–871. https://doi.org/10.1203/00006450-199605000-00021
24. Wiegman, A., Hutten, B. A., de Groot, E., Rodenburg, J., Bakker, H. D., Büller, H. R., Sijbrands, E. J., & Kastelein, J. J. (2004). Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA, 292(3), 331–337.
https://doi.org/10.1001/jama.292.3.331
25. de Jongh, S., Lilien, M. R., op't Roodt, J., Stroes, E. S., Bakker, H. D., & Kastelein, J. J. (2002). Early statin therapy restores endothelial function in children with familial hypercholesterolemia. Journal of the American College of Cardiology, 40(12), 2117–2121.
https://doi.org/10.1016/s0735-1097(02)02593-7
26. Couture, P., Brun, L. D., Szots, F., Lelièvre, M., Gaudet, D., Després, J. P., Simard, J., Lupien, P. J., & Gagné, C. (1998). Association of specific LDL receptor gene mutations with differential plasma lipoprotein response to simvastatin in young French Canadians with heterozygous familial hypercholesterolemia. Arteriosclerosis, thrombosis, and vascular biology, 18(6), 1007–1012. https://doi.org/10.1161/01.atv.18.6.1007
27. McCrindle, B. W., Ose, L., & Marais, A. D. (2003). Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial. The Journal of pediatrics, 143(1), 74–80. https://doi.org/10.1016/S0022-3476(03)00186-0
28. Langslet, G., Breazna, A., & Drogari, E. (2016). A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia. Journal of clinical lipidology, 10(5), 1153–1162.e3. https://doi.org/10.1016/j.jacl.2016.05.010
29. Adams SP, Alaeiilkhchi N, Wright JM. Pitavastatin for lowering lipids. Cochrane Database of Systematic Reviews 2020, Issue 6. Art. No.: CD012735.
DOI: 10.1002/14651858.CD012735.pub2. Accessed 23 February 2023.
30. Harada-Shiba, M., Kastelein, J. J. P., Hovingh, G. K., Ray, K. K., Ohtake, A., Arisaka, O., Ohta, T., Okada, T., Suganami, H., & Wiegman, A. (2018). Efficacy and Safety of Pitavastatin in Children and Adolescents with Familial Hypercholesterolemia in Japan and Europe. Journal of atherosclerosis and thrombosis, 25(5), 422–429. https://doi.org/10.5551/jat.42242
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https://doi.org/10.1016/j.jacc.2009.10.042
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Jul 15, 2023
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How to Cite
Héctor E Arriaga-Cázares, Carlos Fernando Crespo De Vega, Sergio Charles Lozoya, David Vega Morales, & Alexandro J Martagón. (2023). STATIN TREATMENT AND POTENCY ROLE IN CHILDREN WITH FAMILIAL HYPERCHOLESTEROLEMIA: A REVIEW OF EFFICACY AND SAFETY. Journal of Population Therapeutics and Clinical Pharmacology, 30(17), 228–235. https://doi.org/10.53555/jptcp.v30i17.2363
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Author Biographies
Héctor E Arriaga-Cázares
Tecnológico de Monterrey, Escuela de Medicina y Ciencias de la Salud, Monterrey, Nuevo León, México
Carlos Fernando Crespo De Vega
Tecnológico de Monterrey, Escuela de Medicina y Ciencias de la Salud, Monterrey, Nuevo León, México.
Sergio Charles Lozoya
Instituto Mexicano del Seguro Social. UMAE Hospital de Traumatología y Ortopedia No. 21, Nuevo León, México
David Vega Morales
Instituto Mexicano del Seguro Social. Hospital General de Zona No. 17, Nuevo León, México.
Alexandro J Martagón
Tecnológico de Monterrey, Escuela de Medicina y Ciencias de la Salud, Monterrey, Nuevo León, México.