Clinical trial design principles and endpoint definitions for transcatheter mitral valve repair and replacement: part 2: endpoint definitions: A consensus document from the Mitral Valve Academic Research Consortium

Eur Heart J. 2015 Aug 1;36(29):1878-91. doi: 10.1093/eurheartj/ehv333. Epub 2015 Jul 13.

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 trans- catheter 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.

Keywords: Heart failure; Mitral regurgitation; Mitral valve; Valve intervention; Valve surgery (or cardiac surgery).

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Acute Kidney Injury / etiology
  • Acute Kidney Injury / mortality
  • Arrhythmias, Cardiac / etiology
  • Arrhythmias, Cardiac / mortality
  • Cardiac Catheterization / adverse effects
  • Cardiac Catheterization / methods*
  • Cardiac Catheterization / mortality
  • Clinical Trials as Topic / methods*
  • Consensus
  • Endpoint Determination
  • Heart Failure / etiology
  • Heart Failure / mortality
  • Heart Valve Prosthesis
  • Heart Valve Prosthesis Implantation / adverse effects
  • Heart Valve Prosthesis Implantation / methods*
  • Heart Valve Prosthesis Implantation / mortality
  • Hospitalization
  • Humans
  • Mitral Valve Insufficiency / mortality
  • Mitral Valve Insufficiency / surgery*
  • Multidetector Computed Tomography / methods
  • Myocardial Infarction / etiology
  • Myocardial Infarction / mortality
  • Postoperative Hemorrhage / etiology
  • Postoperative Hemorrhage / mortality
  • Prosthesis Failure
  • Risk Assessment
  • Stroke / etiology
  • Stroke / mortality
  • Treatment Outcome