This observational study will provide data (variations in ventricular size and function) that are essential to designing and conducting clinical trials. In addition, the study will evaluate intra- and inter-study variability seen in echocardiography.

BACKGROUND:

Cardiomyopathy is an important cause of chronic disability and death in pediatric patients and currently accounts for approximately 50% of cardiac transplants performed during childhood. Left ventricular (LV) size and function are important independent predictors of outcome, and echocardiography is the primary way to assess ventricular function in children. Although there is extensive experience with this technology, data are limited on how ventricular function changes over time in children, which is a major impediment to conducting controlled trials of therapy in children.

This study has been approved by the Institutional Review Boards/Research Ethics Boards of all participating clinical centers:

Hospital for Sick Children, Toronto, Canada

Children's Hospital Boston, Boston, MA

Columbia College of Physicians and Surgeons, New York, NY

Children's Hospital of Philadelphia, Philadelphia, PA

Duke University Medical Center, Durham, NC

Brody School of Medicine at East Carolina University, Greenville, NC

Wake Forest Baptist Medical Center, Winston Salem, NC

Medical University of South Carolina, Charleston, SC

Primary Children's Medical Center, Salt Lake City, UT

Washington University, St. Louis, MO

DESIGN NARRATIVE:

This observational study will prospectively evaluate pediatric patients with dilated cardiomyopathy who are undergoing clinically indicated echocardiographic evaluation of LV function. Patients who undergo at least two echocardiograms 3 to 18 months apart will have their studies performed by the same ultrasonographer. All studies will be sent to the core laboratory for evaluation of variability in LV mass, volume, and ejection fraction.

Inclusion Criteria:

• Less than 22 years of age
• Any race
• Diagnosis of dilated cardiomyopathy
• LV and diastolic diameter greater than 5.5 cm (or z-score for BSA greater than 2) on the primary image acquisition from the first study echocardiogram
• LV ejection fraction less than 50% (or z-score for age less than -2) or shortening fraction less than 28% (or z-score for age less than -2) as measured on the primary image acquisition from the first study echocardiogram
• Disease onset greater than 2 months prior to screening
• Anticipated to undergo repeat evaluation at the same institution at least 3 months but not more than 13 months later
• Informed consent of parent(s) or legal guardian and assent of subject if required

Exclusion Criteria:

• Hypertrophic cardiomyopathy
• Restrictive cardiomyopathy
• Myocardial noncompaction (LV hypertrabeculation); patient is eligible for the study as long as the echocardiogram performed at the time of screening has no evidence of myocardial noncompaction
• Ventricular paced rhythm
• Atrial or ventricular ectopy at ratio greater than 1:4
• Suspected acute myocarditis
• Tachycardia-induced cardiomyopathy
• Congenital heart disease (repaired or unrepaired)
• Currently on intravenous inotropic support
• Current left ventricular assist device (LVAD) or extracorporeal membrane oxygenation (ECMO)
• Heart transplant waiting list status of 1A or 1B
• Co-morbid condition that precludes the ability to successfully obtain an echocardiogram according to the specifications of the study protocol

Patients up to 22 years of age with dilated cardiomyopathy followed at participating clinical centers or newly diagnosed during the recruitment period