Clinical Trial Design Principles and Endpoint Definitions for Transcatheter Mitral Valve Repair and Replacement: Part 1: Clinical Trial Design Principles A Consensus Document from the Mitral Valve Academic Research Consortium

Mitral Valve Academic Research Consortium (MVARC)

Research output: Contribution to journalReview article

57 Citations (Scopus)

Abstract

Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous etiologies, 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 remodeling 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)278-307
Number of pages30
JournalJournal of the American College of Cardiology
Volume66
Issue number3
DOIs
StatePublished - Jul 21 2015

Fingerprint

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

All Science Journal Classification (ASJC) codes

  • Cardiology and Cardiovascular Medicine

Cite this

@article{4d34949e182e4a10846b130f3da5dcf0,
title = "Clinical Trial Design Principles and Endpoint Definitions for Transcatheter Mitral Valve Repair and Replacement: Part 1: Clinical Trial Design Principles A Consensus Document from the Mitral Valve Academic Research Consortium",
abstract = "Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous etiologies, 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 remodeling 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.",
author = "{Mitral Valve Academic Research Consortium (MVARC)} and Stone, {Gregg W.} and Vahanian, {Alec S.} and Adams, {David H.} and Abraham, {William T.} and Borer, {Jeffrey S.} and Bax, {Jeroen J.} and Joachim Schofer and Cutlip, {Donald E.} and Krucoff, {Mitchell W.} and Blackstone, {Eugene H.} and Philippe Genereux and Mack, {Michael J.} and Siegel, {Robert J.} and Grayburn, {Paul A.} and Maurice Enriquez-Sarano and Patrizio Lancellotti and Gerasimos Filippatos and Kappetein, {Arie Pieter}",
year = "2015",
month = "7",
day = "21",
doi = "10.1016/j.jacc.2015.05.046",
language = "English (US)",
volume = "66",
pages = "278--307",
journal = "Journal of the American College of Cardiology",
issn = "0735-1097",
publisher = "Elsevier USA",
number = "3",

}

TY - JOUR

T1 - Clinical Trial Design Principles and Endpoint Definitions for Transcatheter Mitral Valve Repair and Replacement

T2 - Part 1: Clinical Trial Design Principles A Consensus Document from the Mitral Valve Academic Research Consortium

AU - Mitral Valve Academic Research Consortium (MVARC)

AU - Stone, Gregg W.

AU - Vahanian, Alec S.

AU - Adams, David H.

AU - Abraham, William T.

AU - Borer, Jeffrey S.

AU - Bax, Jeroen J.

AU - Schofer, Joachim

AU - Cutlip, Donald E.

AU - Krucoff, Mitchell W.

AU - Blackstone, Eugene H.

AU - Genereux, Philippe

AU - Mack, Michael J.

AU - Siegel, Robert J.

AU - Grayburn, Paul A.

AU - Enriquez-Sarano, Maurice

AU - Lancellotti, Patrizio

AU - Filippatos, Gerasimos

AU - Kappetein, Arie Pieter

PY - 2015/7/21

Y1 - 2015/7/21

N2 - Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous etiologies, 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 remodeling 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.

AB - Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous etiologies, 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 remodeling 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.

UR - http://www.scopus.com/inward/record.url?scp=84937410219&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84937410219&partnerID=8YFLogxK

U2 - 10.1016/j.jacc.2015.05.046

DO - 10.1016/j.jacc.2015.05.046

M3 - Review article

C2 - 26184622

AN - SCOPUS:84937410219

VL - 66

SP - 278

EP - 307

JO - Journal of the American College of Cardiology

JF - Journal of the American College of Cardiology

SN - 0735-1097

IS - 3

ER -