Current and emerging monoclonal antibodies for treating familial hypercholesterolemia in children

References

• Wiegman A, Gidding SS, Watts GF, et al. Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment. Eur Heart J. 2015;36(36):2425–2437. doi: 10.1093/eurheartj/ehv157
   PubMed Web of Science ®Google Scholar
• Beheshti SO, Madsen CM, Varbo A, et al. Worldwide prevalence of familial hypercholesterolemia: meta-analyses of 11 million subjects. J Am Coll Cardiol. 2020;75(20):2553–2566. doi: 10.1016/j.jacc.2020.03.057
   PubMed Web of Science ®Google Scholar
• Nordestgaard BG, Chapman MJ, Humphries SE, et al. Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European atherosclerosis society. Eur Heart J. 2013;34(45):3478–3490a. doi: 10.1093/eurheartj/eht273
   PubMed Web of Science ®Google Scholar
• Ademi Z, Norman R, Pang J, et al. Cost-effectiveness and return on investment of a nationwide case-finding program for familial hypercholesterolemia in children in the Netherlands. JAMA Pediatr. 2023;177(6):625–632. doi: 10.1001/jamapediatrics.2023.0763
   PubMed Web of Science ®Google Scholar
• Cuchel M, Raal FJ, Hegele RA, et al. Update on European atherosclerosis society consensus statement on homozygous familial hypercholesterolaemia: new treatments and clinical guidance. Eur Heart J. 2023;44(25):2277–2291.doi: 10.1093/eurheartj/ehad197
   PubMed Web of Science ®Google Scholar
• Reiner Ž. Treatment of children with homozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2020;25(10):1095–1097. 10.1177/2047487318781360
   Web of Science ®Google Scholar
• Abifadel M, Boileau C. Genetic and molecular architecture of familial hypercholesterolemia. J Intern Med. 2023;293(2):144–165. doi: 10.1111/joim.13577
   PubMed Web of Science ®Google Scholar
• Shah N, Ahmed H, Tang W. Familial hypercholesterolemia: detect, treat, and ask about family. Cleve Clin J Med. 2020;87(2):109–120. doi: 10.3949/ccjm.87a.19021
   PubMed Web of Science ®Google Scholar
• Peterson AS, Fong LG, Young SG. PCSK9 function and physiology. J Lipid Res. 2008;49(6):1152–1156. doi: 10.1194/jlr.E800008-JLR200
   PubMed Web of Science ®Google Scholar
• Reijman MD, Kusters DM, Wiegman A. Advances in familial hypercholesterolaemia in children. Lancet Child Adolesc Health. 2021;5(9):652–661. doi: 10.1016/S2352-4642(21)00095-X
   PubMedGoogle Scholar
• Saltijeral A, Pérez de Isla L, Alonso R, et al. Attainment of LDL cholesterol treatment goals in children and adolescents with familial hypercholesterolemia. The SAFEHEART follow-up registry. Rev Esp Cardiol (Engl Ed). 2017;70(6):444–450. doi: 10.1016/j.rec.2016.10.010
   PubMed Web of Science ®Google Scholar
• Bigossi M, Maroteau C, Dawed AY, et al. A gene risk score using missense variants in SLCO1B1 is associated with earlier onset statin intolerance. Eur Heart J Cardiovasc Pharmacother. 2023;9(6):536–545. doi: 10.1093/ehjcvp/pvad040
   PubMed Web of Science ®Google Scholar
• Link E, Parish S, Armitage J, et al. SLCO1B1 variants and statin-induced myopathy–a genomewide study. N Engl J Med. 2008;359(8):789–799.
   PubMed Web of Science ®Google Scholar
• Behring K. Ueber das Zustandekommen der Diphtherie-Immunität und der Tetanus-Immunität bei Thieren. Dtsch Med Wochenschr. 1965;16(49):1113–1114.
   Google Scholar
• Köhler G, Milstein C. Continuous cultures of fused cells secreting antibody of predefined specificity. Nature. 1975;256(5517):495–497. doi: 10.1038/256495a0
   PubMed Web of Science ®Google Scholar
• Ito MK, Santos RD. PCSK9 inhibition with monoclonal antibodies: modern management of hypercholesterolemia. J Clin Pharmacol. 2017;57(1):7–32. doi: 10.1002/jcph.766
   PubMed Web of Science ®Google Scholar
• Farnier M. PCSK9: from discovery to therapeutic applications. Arch Cardiovasc Dis. 2014;107(1):58–66. doi: 10.1016/j.acvd.2013.10.007
   PubMed Web of Science ®Google Scholar
• Page MM, Watts GF PCSK9 inhibitors – mechanisms of action. Aust Prescr 2016;39:164–167. 5 doi: 10.18773/austprescr.2016.060
   PubMed Web of Science ®Google Scholar
• Santos RD, Ruzza A, Hovingh GK, et al. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020;383(14):1317–1327. doi: 10.1056/NEJMoa2019910
   PubMed Web of Science ®Google Scholar
• Guedeney P, Giustino G, Sorrentino S, et al. Efficacy and safety of alirocumab and evolocumab: a systematic review and meta-analysis of randomized controlled trials. Eur Heart J. 2019;43(7):e17–e25. doi: 10.1093/eurheartj/ehz430
   Google Scholar
• Ge X, Zhu T, Zeng H, et al. A systematic review and meta-analysis of therapeutic efficacy and safety of Alirocumab and evolocumab on familial hypercholesterolemia. Biomed Res Int. 2021;2021:8032978. doi: 10.1155/2021/8032978
   PubMed Web of Science ®Google Scholar
• Santos RD, Ruzza A, Hovingh GK, et al. Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT. Lancet Diabetes Endocrinol. 2022;10(10):732–740. doi: 10.1016/S2213-8587(22)00221-2
   PubMed Web of Science ®Google Scholar
• Santos RD, Ruzza A, Wang B, et al. Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment. Eur J Prev Cardiol. 2023;31(3):1–9. doi: 10.1093/eurjpc/zwad332
   Web of Science ®Google Scholar
• Santos R, Wiegman A, Caprio S, et al. Efficacy and safety of alirocumab in children and adolescents with heterozygous familial hypercholesterolaemia inadequately controlled with statins. Eur Heart J. 2023;44(Supplement_2). doi: 10.1093/eurheartj/ehad655.2809
   Google Scholar
• Lin J, Chen J, Zhang R, et al. Abstract 10921: efficacy and safety of ongericimab in Chinese patients with homozygous familial hypercholesterolemia. Circulation. 2022;146(Suppl_1):A10921–A10921. doi: 10.1161/circ.146.suppl_1.10921
   Google Scholar
• Raal FJ, Rosenson RS, Reeskamp LF, et al. Evinacumab for Homozygous Familial Hypercholesterolemia. N Engl J Med. 2020;383(8):711–720. doi: 10.1056/NEJMoa2004215
   PubMed Web of Science ®Google Scholar
• Wiegman A, Greber-Platzer S, Ali S, et al. Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia. Circulation. 2023;149(5):343–353. doi: 10.1161/CIRCULATIONAHA.123.065529
   PubMed Web of Science ®Google Scholar
• Rosenson RS, Burgess LJ, Ebenbichler CF, et al. Evinacumab in patients with refractory hypercholesterolemia. N Engl J Med. 2020;383(24):2307–2319. doi: 10.1056/NEJMoa2031049
   PubMed Web of Science ®Google Scholar
• Korman MJ, Retterstøl K, Kristiansen IS, et al. Are PCSK9 inhibitors cost effective? Pharmaceutics. 2018;36(9):1031–1041. doi: 10.1007/s40273-018-0671-0
   Google Scholar
• Raal FJ, Hovingh GK, Blom D, et al. Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study. Lancet Diabetes Endocrinol. 2017;5(4):280–290. doi: 10.1016/S2213-8587(17)30044-X
   PubMed Web of Science ®Google Scholar
• Wołowiec Ł, Osiak J, Wołowiec A, et al. Inclisiran—safety and effectiveness of small interfering RNA in inhibition of PCSK-9. Pharmaceutics. 2023;15(2). 323 doi: 10.3390/pharmaceutics15020323
   PubMed Web of Science ®Google Scholar
• Ray KK, Wright RS, Kallend D, et al. Two phase 3 trials of inclisiran in patients with elevated LDL cholesterol. N Engl J Med. 2020;382(16):1507–1519. doi: 10.1056/NEJMoa1912387
   PubMed Web of Science ®Google Scholar
• Reijman MD, Schweizer A, Peterson ALH, et al. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022;29(9):1361–1368. doi: 10.1093/eurjpc/zwac025
   PubMed Web of Science ®Google Scholar
• Nissen SE, Lincoff AM, Brennan D, et al. Bempedoic acid and cardiovascular outcomes in statin-intolerant patients. N Engl J Med. 2023;388(15):1353–1364. doi: 10.1056/NEJMoa2215024
   PubMed Web of Science ®Google Scholar