Clinical trial design principles and endpoint definitions for transcatheter mitral valve repair and replacement

Part 1: Clinical trial design principles

Gregg W. Stone, Alec S. Vahanian, David H. Adams, William T. Abraham, Jeffrey S. Borer, Jeroen J. Bax, Joachim Schofer, Donald E. Cutlip, Mitchell W. Krucoff, Eugene H. Blackstone, Philippe GCrossed D Sign Crossed D Signreux, Michael J. Mack, Robert J. Siegel, Paul A. Grayburn, Maurice E Sarano, Patrizio Lancellotti, Gerasimos Filippatos, Arie Pieter Kappetein

Research output: Contribution to journalArticle

16 Citations (Scopus)

Abstract

Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous aetiologies, including primary (organic) MR, due to underlying degenerative/structural mitral valve (MV) pathology, and secondary (functional) MR, which is principally caused by global or regional left ventricular remodelling and/or severe left atrial dilation. Diagnosis and optimal management of MR requires integration of valve disease and heart failure specialists, MV cardiac surgeons, interventional cardiologists with expertise in structural heart disease, and imaging experts. The introduction of transcatheter MV therapies has highlighted the need for a consensus approach to pragmatic clinical trial design and uniform endpoint definitions to evaluate outcomes in patients with MR. The Mitral Valve Academic Research Consortium is a collaboration between leading academic research organizations and physician-scientists specializing in MV disease from the United States and Europe. Three in-person meetings were held in Virginia and New York during which 44 heart failure, valve, and imaging experts, MV surgeons and interventional cardiologists, clinical trial specialists and statisticians, and representatives from the U.S. Food and Drug Administration considered all aspects of MV pathophysiology, prognosis, and therapies, culminating in a 2-part document describing consensus recommendations for clinical trial design (Part 1) and endpoint definitions (Part 2) to guide evaluation of transcatheter and surgical therapies for MR. The adoption of these recommendations will afford robustness and consistency in the comparative effectiveness evaluation of new devices and approaches to treat MR. These principles may be useful for regulatory assessment of new transcatheter MV devices, as well as for monitoring local and regional outcomes to guide quality improvement initiatives.

Original languageEnglish (US)
Pages (from-to)1851-1877
Number of pages27
JournalEuropean Heart Journal
Volume36
Issue number29
DOIs
StatePublished - Aug 1 2015

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Mitral Valve
Mitral Valve Insufficiency
Clinical Trials
Heart Failure
Pragmatic Clinical Trials
Equipment and Supplies
Ventricular Remodeling
Heart Valves
United States Food and Drug Administration
Quality Improvement
Research
Dilatation
Heart Diseases
Therapeutics
Organizations
Pathology
Physicians

Keywords

  • Heart failure
  • Mitral regurgitation
  • Mitral valve
  • Valve intervention
  • Valve surgery (or cardiac surgery)

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Clinical trial design principles and endpoint definitions for transcatheter mitral valve repair and replacement : Part 1: Clinical trial design principles. / Stone, Gregg W.; Vahanian, Alec S.; Adams, David H.; Abraham, William T.; Borer, Jeffrey S.; Bax, Jeroen J.; Schofer, Joachim; Cutlip, Donald E.; Krucoff, Mitchell W.; Blackstone, Eugene H.; GCrossed D Sign Crossed D Signreux, Philippe; Mack, Michael J.; Siegel, Robert J.; Grayburn, Paul A.; Sarano, Maurice E; Lancellotti, Patrizio; Filippatos, Gerasimos; Kappetein, Arie Pieter.

In: European Heart Journal, Vol. 36, No. 29, 01.08.2015, p. 1851-1877.

Research output: Contribution to journalArticle

Stone, GW, Vahanian, AS, Adams, DH, Abraham, WT, Borer, JS, Bax, JJ, Schofer, J, Cutlip, DE, Krucoff, MW, Blackstone, EH, GCrossed D Sign Crossed D Signreux, P, Mack, MJ, Siegel, RJ, Grayburn, PA, Sarano, ME, Lancellotti, P, Filippatos, G & Kappetein, AP 2015, 'Clinical trial design principles and endpoint definitions for transcatheter mitral valve repair and replacement: Part 1: Clinical trial design principles', European Heart Journal, vol. 36, no. 29, pp. 1851-1877. https://doi.org/10.1093/eurheartj/ehv281
Stone, Gregg W. ; Vahanian, Alec S. ; Adams, David H. ; Abraham, William T. ; Borer, Jeffrey S. ; Bax, Jeroen J. ; Schofer, Joachim ; Cutlip, Donald E. ; Krucoff, Mitchell W. ; Blackstone, Eugene H. ; GCrossed D Sign Crossed D Signreux, Philippe ; Mack, Michael J. ; Siegel, Robert J. ; Grayburn, Paul A. ; Sarano, Maurice E ; Lancellotti, Patrizio ; Filippatos, Gerasimos ; Kappetein, Arie Pieter. / Clinical trial design principles and endpoint definitions for transcatheter mitral valve repair and replacement : Part 1: Clinical trial design principles. In: European Heart Journal. 2015 ; Vol. 36, No. 29. pp. 1851-1877.
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