Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial
Tài liệu tham khảo
Marian, 2017, Hypertrophic cardiomyopathy: genetics, pathogenesis, clinical manifestations, diagnosis, and therapy, Circ Res, 121, 749, 10.1161/CIRCRESAHA.117.311059
Maron, 2018, Clinical course and management of hypertrophic cardiomyopathy, N Engl J Med, 379, 655, 10.1056/NEJMra1710575
Seferović, 2019, Heart failure in cardiomyopathies: a position paper from the Heart Failure Association of the European Society of Cardiology, Eur J Heart Fail, 21, 553, 10.1002/ejhf.1461
Sequeira, 2019, Energetic drain driving hypertrophic cardiomyopathy, FEBS Lett, 593, 1616, 10.1002/1873-3468.13496
Ho, 2018, Genotype and lifetime burden of disease in hypertrophic cardiomyopathy: insights from the sarcomeric human cardiomyopathy registry (SHaRe), Circulation, 138, 1387, 10.1161/CIRCULATIONAHA.117.033200
Gersh, 2011, 2011 ACCF/AHA guideline for the diagnosis and treatment of hypertrophic cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines, Circulation, 124, e783
Elliott, 2014, 2014 ESC Guidelines on diagnosis and management of hypertrophic cardiomyopathy: the Task Force for the Diagnosis and Management of Hypertrophic Cardiomyopathy of the European Society of Cardiology (ESC), Eur Heart J, 35, 2733, 10.1093/eurheartj/ehu284
Kaltenbach, 1979, Treatment of hypertrophic obstructive cardiomyopathy with verapamil, Br Heart J, 42, 35, 10.1136/hrt.42.1.35
Cohen, 1967, Amelioration of angina pectoris in idiopathic hypertrophic subaortic stenosis with beta-adrenergic blockade, Circulation, 35, 847, 10.1161/01.CIR.35.5.847
Ammirati, 2016, Pharmacological treatment of hypertrophic cardiomyopathy: current practice and novel perspectives, Eur J Heart Fail, 18, 1106, 10.1002/ejhf.541
Liebregts, 2015, A systematic review and meta-analysis of long-term outcomes after septal reduction therapy in patients with hypertrophic cardiomyopathy, JACC Heart Fail, 3, 896, 10.1016/j.jchf.2015.06.011
Wells, 2018, Clinical profile of nonresponders to surgical myectomy with obstructive hypertrophic cardiomyopathy, Am J Med, 131, e235, 10.1016/j.amjmed.2017.12.031
Kim, 2016, Hospital volume outcomes after septal myectomy and alcohol septal ablation for treatment of obstructive hypertrophic cardiomyopathy: US Nationwide Inpatient Database, 2003–2011, JAMA Cardiol, 1, 324, 10.1001/jamacardio.2016.0252
Grillo, 2019, In vitro and in vivo pharmacokinetic characterization of mavacamten, a first-in-class small molecule allosteric modulator of beta cardiac myosin, Xenobiotica, 49, 718, 10.1080/00498254.2018.1495856
Kawas, 2017, A small-molecule modulator of cardiac myosin acts on multiple stages of the myosin chemomechanical cycle, J Biol Chem, 292, 16571, 10.1074/jbc.M117.776815
Anderson, 2018, Deciphering the super relaxed state of human β-cardiac myosin and the mode of action of mavacamten from myosin molecules to muscle fibers, Proc Natl Acad Sci USA, 115, E8143, 10.1073/pnas.1809540115
del Rio, 2018, In vivo cardiac effects of mavacamten (MYK-461): evidence for negative inotropy and improved compliance, Circulation, 136
Green, 2016, A small-molecule inhibitor of sarcomere contractility suppresses hypertrophic cardiomyopathy in mice, Science, 351, 617, 10.1126/science.aad3456
Heitner, 2019, Mavacamten treatment for obstructive hypertrophic cardiomyopathy: a clinical trial, Ann Intern Med, 170, 741, 10.7326/M18-3016
Ho, 2020, Evaluation of mavacamten in symptomatic patients with nonobstructive hypertrophic cardiomyopathy, J Am Coll Cardiol, 75, 2649, 10.1016/j.jacc.2020.03.064
Ho, 2020, Study design and rationale of EXPLORER-HCM: evaluation of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy, Circ Heart Fail, 13, 10.1161/CIRCHEARTFAILURE.120.006853
Lewis, 2017, Health-related quality of life outcomes in PARADIGM-HF, Circ Heart Fail, 10, 10.1161/CIRCHEARTFAILURE.116.003430
Cowger, 2018, Quality of life and functional capacity outcomes in the MOMENTUM 3 trial at 6 months: a call for new metrics for left ventricular assist device patients, J Heart Lung Transplant, 37, 15, 10.1016/j.healun.2017.10.019
Kubo, 2011, Combined measurements of cardiac troponin I and brain natriuretic peptide are useful for predicting adverse outcomes in hypertrophic cardiomyopathy, Circ J, 75, 919, 10.1253/circj.CJ-10-0782
Geske, 2013, B-type natriuretic peptide and survival in hypertrophic cardiomyopathy, J Am Coll Cardiol, 61, 2456, 10.1016/j.jacc.2013.04.004
Seydelmann, 2016, High-sensitivity troponin: a clinical blood biomarker for staging cardiomyopathy in Fabry disease, J Am Heart Assoc, 5, 10.1161/JAHA.115.002839
Nielen, 2019, The association between β-blocker use and cardiorespiratory fitness: the maastricht study, J Cardiovasc Pharmacol Ther, 24, 37, 10.1177/1074248418778551
Malhotra, 2016, Cardiopulmonary exercise testing in heart failure, JACC Heart Fail, 4, 607, 10.1016/j.jchf.2016.03.022
Myers, 2007, Cardiac output and cardiopulmonary responses to exercise in heart failure: application of a new bio-reactance device, J Card Fail, 13, 629, 10.1016/j.cardfail.2007.05.009