Pragmatic Design of Randomized Clinical Trials for Heart Failure

JACC: Heart Failure - Tập 9 Số 5 - Trang 325-335 - 2021
Stephen J. Greene1, Eric J. Velazquez2, Kevin J. Anstrom3, Eric L. Eisenstein3, Shelly Sapp3, Shelby Morgan3, Tina Harding3, Vandana Sachdev4, Fassil Ketema4, Dong‐Yun Kim4, Patrice Desvigne‐Nickens4, Bertram Pitt5, Robert J. Mentz6
1Duke Clinical Research Institute, Durham, North Carolina, USA; Division of Cardiology, Duke University School of Medicine, Durham, North Carolina, USA. Electronic address: https://twitter.com/SJGreene_md.
2Division of Cardiology, Yale University School of Medicine, New Haven, Connecticut, USA. Electronic address: https://twitter.com/ericjvelazquez.
3Duke Clinical Research Institute, Durham, North Carolina, USA
4Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute, Bethesda, Maryland, USA
5Division of Cardiology, University of Michigan, Ann Arbor, Michigan, USA
6Duke Clinical Research Institute, Durham, North Carolina, USA; Division of Cardiology, Duke University School of Medicine, Durham, North Carolina, USA. Electronic address: [email protected].

Tóm tắt

Từ khóa


Tài liệu tham khảo

Yancy, 2013, 2013 ACCF/AHA guideline for the management of heart failure: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines, J Am Coll Cardiol, 62, e147, 10.1016/j.jacc.2013.05.019

Harinstein, 2015, Site selection for heart failure clinical trials in the USA, Heart Fail Rev, 20, 375, 10.1007/s10741-015-9473-z

Samman Tahhan, 2018, Design elements and enrollment patterns of contemporary trials in heart failure with preserved ejection fraction: a systematic review, J Am Coll Cardiol HF, 6, 714

Butler, 2013, Relationship between clinical trial site enrollment with participant characteristics, protocol completion, and outcomes: insights from the EVEREST (Efficacy of Vasopressin Antagonism in Heart Failure: Outcome Study with Tolvaptan) trial, J Am Coll Cardiol, 61, 571, 10.1016/j.jacc.2012.10.025

Teerlink, 2013, Serelaxin, recombinant human relaxin-2, for treatment of acute heart failure (RELAX-AHF): a randomised, placebo-controlled trial, Lancet, 381, 29, 10.1016/S0140-6736(12)61855-8

Gheorghiade, 2013, Effect of aliskiren on postdischarge mortality and heart failure readmissions among patients hospitalized for heart failure: the ASTRONAUT randomized trial, JAMA, 309, 1125, 10.1001/jama.2013.1954

Packer, 2017, Effect of ularitide on cardiovascular mortality in acute heart failure, N Engl J Med, 376, 1956, 10.1056/NEJMoa1601895

Metra, 2019, Effects of serelaxin in patients with acute heart failure, N Engl J Med, 381, 716, 10.1056/NEJMoa1801291

Summer, 2012, Physician perception of the impact of productivity measures on academic practice, Arch Intern Med, 172, 967, 10.1001/archinternmed.2012.1579

Greene, 2016, Influence of clinical trial site enrollment on patient characteristics, protocol completion, and end points: insights from the ASCEND-HF trial (Acute Study of Clinical Effectiveness of Nesiritide in Decompensated Heart Failure), Circ Heart Fail, 9, 10.1161/CIRCHEARTFAILURE.116.002986

Greene, 2019, Representativeness of a heart failure trial by race and sex: results from ASCEND-HF and GWTG-HF, J Am Coll Cardiol HF, 7, 980

Tahhan, 2018, Enrollment of older patients, women, and racial and ethnic minorities in contemporary heart failure clinical trials: a systematic review, JAMA Cardiol, 3, 1011, 10.1001/jamacardio.2018.2559

Martin, 2017, How much do clinical trials cost?, Nat Rev Drug Discov, 16, 381, 10.1038/nrd.2017.70

Moore, 2018, Estimated costs of pivotal trials for novel therapeutic agents approved by the US Food and Drug Administration, 2015–2016, JAMA Intern Med, 178, 1451, 10.1001/jamainternmed.2018.3931

Samman Tahhan, 2018, Evolving landscape of clinical trials in heart failure: patient populations, endpoint selection, and regions of enrollment, Curr Heart Fail Rep, 15, 10, 10.1007/s11897-018-0374-z

Psotka, 2019, Heart failure site-based research in the United States: results of the Heart Failure Society of America Research Network Survey, J Am Coll Cardiol HF, 7, 431

Loudon, 2015, The PRECIS-2 tool: designing trials that are fit for purpose, BMJ, 350, h2147, 10.1136/bmj.h2147

Mentz, 2016, Good clinical practice guidance and pragmatic clinical trials: balancing the best of both worlds, Circulation, 133, 872, 10.1161/CIRCULATIONAHA.115.019902

Federspiel, 2016, Comparing inverse probability of treatment weighting and instrumental variable methods for the evaluation of adenosine diphosphate receptor inhibitors after percutaneous coronary intervention, JAMA Cardiol, 1, 655, 10.1001/jamacardio.2016.1783

Hernandez, 2012, Associations between aldosterone antagonist therapy and risks of mortality and readmission among patients with heart failure and reduced ejection fraction, JAMA, 308, 2097, 10.1001/jama.2012.14795

Buggey, 2015, A reappraisal of loop diuretic choice in heart failure patients, Am Heart J, 169, 323, 10.1016/j.ahj.2014.12.009

Greene, 2018, Potential advantages of torsemide in patients with heart failure: more than just a 'water pill'?, Eur J Heart Fail, 20, 471, 10.1002/ejhf.1024

Kasama, 2006, Effects of torasemide on cardiac sympathetic nerve activity and left ventricular remodelling in patients with congestive heart failure, Heart, 92, 1434, 10.1136/hrt.2005.079764

Lopez, 2004, Effects of loop diuretics on myocardial fibrosis and collagen type I turnover in chronic heart failure, J Am Coll Cardiol, 43, 2028, 10.1016/j.jacc.2003.12.052

Lopez, 2007, Identification of a potential cardiac antifibrotic mechanism of torasemide in patients with chronic heart failure, J Am Coll Cardiol, 50, 859, 10.1016/j.jacc.2007.04.080

Murray, 2001, Open-label randomized trial of torsemide compared with furosemide therapy for patients with heart failure, Am J Med, 111, 513, 10.1016/S0002-9343(01)00903-2

Cosin, 2002, Torasemide in chronic heart failure: results of the TORIC study, Eur J Heart Fail, 4, 507, 10.1016/S1388-9842(02)00122-8

Muller, 2003, Torasemide vs. furosemide in primary care patients with chronic heart failure NYHA II to IV--efficacy and quality of life, Eur J Heart Fail, 5, 793, 10.1016/S1388-9842(03)00150-8

Greene, 2018, Reassessing the role of surrogate end points in drug development for heart failure, Circulation, 138, 1039, 10.1161/CIRCULATIONAHA.118.034668

Bikdeli, 2013, Dominance of furosemide for loop diuretic therapy in heart failure: time to revisit the alternatives?, J Am Coll Cardiol, 61, 1549, 10.1016/j.jacc.2012.12.043

Lachin, 1999, Worst-rank score analysis with informatively missing observations in clinical trials, Control Clin Trials, 20, 408, 10.1016/S0197-2456(99)00022-7

Bang, 2000, Estimating medical costs with censored data, Biometrika, 87, 329, 10.1093/biomet/87.2.329

Wijeysundera, 2009, Bayesian statistical inference enhances the interpretation of contemporary randomized controlled trials, J Clin Epidemiol, 62, 13, 10.1016/j.jclinepi.2008.07.006

Greene, 2018, Relationship between timing of trial randomization, protocol completion, and clinical outcomes among patients hospitalized for heart failure: from the ASTRONAUT trial, Eur J Heart Fail, 20, 1760, 10.1002/ejhf.1310

Alvarez-Garcia, 2020, Prognostic impact of prior heart failure in patients hospitalized with COVID-19, J Am Coll Cardiol, 76, 2334, 10.1016/j.jacc.2020.09.549

Greene, 2015, The vulnerable phase after hospitalization for heart failure, Nat Rev Cardiol, 12, 220, 10.1038/nrcardio.2015.14