Long-Term Evolocumab in Patients With Familial Hypercholesterolemia

Soutar, 2007, Mechanisms of disease: genetic causes of familial hypercholesterolemia, Nat Clin Pract Cardiovasc Med, 4, 214, 10.1038/ncpcardio0836 Foody, 2016, Familial hypercholesterolemia/autosomal dominant hypercholesterolemia: molecular defects, the LDL-C continuum, and gradients of phenotypic severity, J Clin Lipidol, 10, 970, 10.1016/j.jacl.2016.04.009 Rader, 2003, Monogenic hypercholesterolemia: new insights in pathogenesis and treatment, J Clin Invest, 111, 1795, 10.1172/JCI200318925 Cuchel, 2014, Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society, Eur Heart J, 35, 2146, 10.1093/eurheartj/ehu274 de Ferranti, 2016, Prevalence of familial hypercholesterolemia in the 1999 to 2012 United States National Health and Nutrition Examination Surveys (NHANES), Circulation, 133, 1067, 10.1161/CIRCULATIONAHA.115.018791 Catapano, 2016, 2016 ESC/EAS guidelines for the management of dyslipidaemias, Eur Heart J, 37, 2999, 10.1093/eurheartj/ehw272 Gagné, 2002, Efficacy and safety of ezetimibe coadministered with atorvastatin or simvastatin in patients with homozygous familial hypercholesterolemia, Circulation, 105, 2469, 10.1161/01.CIR.0000018744.58460.62 Neil, 2008, Reductions in all-cause, cancer, and coronary mortality in statin-treated patients with heterozygous familial hypercholesterolaemia: a prospective registry study, Eur Heart J, 29, 2625, 10.1093/eurheartj/ehn422 Grundy, 2019, 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA guideline on the management of blood cholesterol. A report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines, J Am Coll Cardiol, 73, 3168, 10.1016/j.jacc.2018.11.002 Gidding, 2015, The agenda for familial hypercholesterolemia. A scientific statement from the American Heart Association, Circulation, 132, 2167, 10.1161/CIR.0000000000000297 Perez de Isla, 2016, Attainment of LDL-cholesterol treatment goals in patients with familial hypercholesterolemia: 5-year SAFEHEART registry follow-up, J Am Coll Cardiol, 67, 1278, 10.1016/j.jacc.2016.01.008 Cuchel, 2013, Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study, Lancet, 381, 40, 10.1016/S0140-6736(12)61731-0 Raal, 2010, Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial, Lancet, 375, 998, 10.1016/S0140-6736(10)60284-X Wang, 2016, Systematic review of low-density lipoprotein cholesterol apheresis for the treatment of familial hypercholesterolemia, J Am Heart Assoc, 5 Kroon, 2000, The rebound of lipoproteins after LDL-apheresis. Kinetics and estimation of mean lipoprotein levels, Atherosclerosis, 152, 519, 10.1016/S0021-9150(00)00371-3 Langslet, 2015, Evolocumab (AMG 145) for primary hypercholesterolemia, Expert Rev Cardiovasc Ther, 13, 477, 10.1586/14779072.2015.1030395 Raal, 2015, Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial, Lancet, 385, 341, 10.1016/S0140-6736(14)61374-X Raal, 2015, PCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trial, Lancet, 385, 331, 10.1016/S0140-6736(14)61399-4 Raal, 2017, 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, 5, 280, 10.1016/S2213-8587(17)30044-X Sabatine, 2017, Evolocumab and clinical outcomes in patients with cardiovascular disease, N Engl J Med, 376, 1713, 10.1056/NEJMoa1615664 Defesche, 2017, Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia, J Clin Lipidol, 11, 1338, 10.1016/j.jacl.2017.08.016 Stein, 2016, PCSK9: the critical role of familial hypercholesterolemia from discovery to benefit for all, Cardiovasc Drugs Ther, 30, 427, 10.1007/s10557-016-6690-1 Nanchen, 2016, Prognosis of patients with familial hypercholesterolemia after acute coronary syndromes, Circulation, 134, 698, 10.1161/CIRCULATIONAHA.116.023007 Perak, 2016, Long-term risk of atherosclerotic cardiovascular disease in US adults with the familial hypercholesterolemia phenotype, Circulation, 134, 9, 10.1161/CIRCULATIONAHA.116.022335 Thompson, 2018, Survival in homozygous familial hypercholesterolaemia is determined by the on-treatment level of serum cholesterol, Eur Heart J, 39, 1162, 10.1093/eurheartj/ehx317 Baum, 2019, Effect of evolocumab on lipoprotein apheresis requirement and lipid levels: results of the randomized, controlled, open-label DE LAVAL study, J Clin Lipidol, 13, 901, 10.1016/j.jacl.2019.10.003 Moriarty, 2016, Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trial, Eur Heart J, 37, 3588, 10.1093/eurheartj/ehw388 Gaudet, 2017, ANGPTL3 inhibition in homozygous familial hypercholesterolemia, N Engl J Med, 377, 296, 10.1056/NEJMc1705994 Gaudet, 2018, Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: rationale and design of the HAUSER-RCT study, J Clin Lipidol, 12, 1199, 10.1016/j.jacl.2018.05.007