title: 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.
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.
authors.affiliation: Columbia University Medical Center/New York-Presbyterian Hospital, New York, New York; Cardiovascular Research Foundation, New York, New York. Electronic address: gs2184@columbia.edu.
authors.forename: Gregg W
authors.initials: GW
authors.lastname: Stone
authors.affiliation: Mount Sinai Health System, New York, New York.
authors.forename: David H
authors.initials: DH
authors.lastname: Adams
authors.affiliation: The Ohio State University, Columbus, Ohio.
authors.forename: William T
authors.initials: WT
authors.lastname: Abraham
authors.affiliation: Erasmus University Medical Center, Rotterdam, the Netherlands.
authors.forename: Arie Pieter
authors.initials: AP
authors.lastname: Kappetein
authors.affiliation: Columbia University Medical Center/New York-Presbyterian Hospital, New York, New York; Cardiovascular Research Foundation, New York, New York; Hôpital du Sacré-Coeur de Montréal, Montreal, Quebec, Canada.
authors.forename: Philippe
authors.initials: P
authors.lastname: Généreux
authors.affiliation: Hartcentrum Hasselt, Hasselt, Belgium.
authors.forename: Pascal
authors.initials: P
authors.lastname: Vranckx
authors.affiliation: Cardiovascular Research Foundation, New York, New York; Mount Sinai Health System, New York, New York.
authors.forename: Roxana
authors.initials: R
authors.lastname: Mehran
authors.affiliation: Asklepios Hospital St. Georg, Hamburg, Germany.
authors.forename: Karl-Heinz
authors.initials: KH
authors.lastname: Kuck
authors.affiliation: Columbia University Medical Center/New York-Presbyterian Hospital, New York, New York; Cardiovascular Research Foundation, New York, New York.
authors.forename: Martin B
authors.initials: MB
authors.lastname: Leon
authors.affiliation: McGill University Health Center, Montreal, Quebec, Canada.
authors.forename: Nicolo
authors.initials: N
authors.lastname: Piazza
authors.affiliation: Erasmus University Medical Center, Rotterdam, the Netherlands.
authors.forename: Stuart J
authors.initials: SJ
authors.lastname: Head
authors.affiliation: Athens University Hospital Attikon, Athens, Greece.
authors.forename: Gerasimos
authors.initials: G
authors.lastname: Filippatos
authors.affiliation: Hôpital Bichat, Paris, France.
authors.forename: Alec S
authors.initials: AS
authors.lastname: Vahanian
mesh_terms: D006328:Cardiac Catheterization / Q000379:methods*; D002986:Clinical Trials as Topic; D019468:Disease Management; D004452:Echocardiography; D023381:Endpoint Determination; D005060:Europe; D019918:Heart Valve Prosthesis Implantation / Q000009:adverse effects / Q000295:instrumentation / Q000379:methods; D006801:Humans; D008943:Mitral Valve / Q000601:surgery*; D008944:Mitral Valve Insufficiency / Q000175:diagnosis / Q000209:etiology / Q000503:physiopathology / Q000601:surgery; D018579:Patient Selection; D011379:Prognosis; D012107:Research Design; D018570:Risk Assessment / Q000379:methods; D014481:United States; D020257:Ventricular Remodeling
keywords: heart failure; mitral regurgitation; mitral valve; valve intervention; valve surgery (or cardiac surgery)