TY  - BOOK
AU  - Epstein, Stephen E
TI  - Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy
SN  - 1558-2027
PY  - 2017///
KW  - *Cardiomyopathies/su [Surgery]
KW  - *Heart Failure/su [Surgery]
KW  - *Mesenchymal Stem Cell Transplantation
KW  - Cardiomyopathies/dg [Diagnostic Imaging]
KW  - Cardiomyopathies/pp [Physiopathology]
KW  - Clinical Protocols
KW  - Cross-Over Studies
KW  - Echocardiography
KW  - Heart Failure/dg [Diagnostic Imaging]
KW  - Heart Failure/pp [Physiopathology]
KW  - Humans
KW  - Magnetic Resonance Imaging, Cine
KW  - Mesenchymal Stem Cell Transplantation/ae [Adverse Effects]
KW  - Myocardium/pa [Pathology]
KW  - Pilot Projects
KW  - Recovery of Function
KW  - Research Design
KW  - Single-Blind Method
KW  - Stroke Volume
KW  - Time Factors
KW  - Treatment Outcome
KW  - United States
KW  - Ventricular Function, Left
KW  - Ventricular Remodeling
KW  - MedStar Health Research Institute
KW  - Journal Article
N1  - Available online through MWHC library: 2006 - present
N2  - AIMS: This article describes an ongoing study investigating the safety and efficacy of ischemia-tolerant mesenchymal stem cell (MSC) therapy in patients with nonischemic heart failure and dysfunctional viable myocardium without scarring. This study will follow principles of the previously described mechanistic translational-phase concept whereby the effect of the study agent on laboratory and imaging markers of cardiac structure and function will be tested in a small homogenous cohort with the goal to enhance the understanding of the effect of interventions on cardiac remodeling and performance; CONCLUSION: This pilot study will explore the safety and effects on cardiac structure and function of i.v. injection of ischemia-tolerant MSCs in a small homogenous cohort of nonischemic heart failure patients with reduced ejection fraction and absent replacement scarring on CMR. This study also represents a prospective mechanistic translational-phase study using baseline and serial CMR imaging in heart failure patients and serves as a potential model for design of future heart failure trials (ClinicalTrials.gov identifier: NCT02467387); STUDY DESIGN: This single-blind, placebo-controlled, crossover, multicenter, randomized study will assess the safety, tolerability, and preliminary efficacy of a single intravenous (i.v.) dose of allogeneic ischemia-tolerant MSCs in individuals with heart failure of nonischemic cause, ejection fraction 40% or less, and dysfunctional viable myocardium who have been receiving guideline-directed medical therapy. Eligible patients will have no evidence of baseline replacement scarring on delayed-enhancement cardiac magnetic resonance (CMR). Approximately 20 patients will be randomized in a 1 : 1 ratio to receive an i.v. infusion of ischemia-tolerant MSCs or placebo. At 90 days, the two groups will undergo crossover and received the alternative treatment. The primary endpoint is safety, as evaluated through at least 1-year post-MSC infusion. Additional efficacy endpoints will include measures of cardiac structure and function, as evaluated by serial cine-CMR and transthoracic echocardiography at 90 and 180 days post-initial infusion
UR  - https://dx.doi.org/10.2459/JCM.0000000000000303
ER  -