Cardiomyopathy (VVV) Study (Observational)

Cardiomyopathy (VVV) Publications


  • Left Ventricular Dysfunction is Associated with Intraventricular Dyssynchrony by 3-Dimensional Echocardiography in Children. G. Baker, J Am Soc Enchocardiogr 2008; 21(3):230-233.
    “Dyssynchrony” means that parts of the heart do not squeeze at the same time. This causes less efficient ejection of blood and contributes to heart failure. There is little data on dyssynchrony in children. This small study looked at whether 3-dimensional echocardiography (heart ultrasound) can be used to identify dyssynchrony in healthy children and children with poor heart function. The results show that 3D echocardiography can be used to look at dyssynchrony in children, and that patients with poorer heart function have more dyssynchrony.

Collaborative Learning

Collaborative Learning Publications

Echo Z-Score

Fontan Study (Observational)

Fontan Publications

Ancillary Studies:
  • Sustained Effects of Cardiac Rehabilitation in Children with Serious Congenital Heart Disease. J. Rhodes, Pediatrics 2006; 118(3):e586-593.
    Previous studies have shown immediate benefits when children with congenital heart disease are enrolled in a cardiac rehabilitation (exercise) program. This study aimed to see if the benefits lasted after the exercise program ended. The results showed, compared with children who did not undergo cardiac rehabilitation after the Fontan procedure, children who did participate had significant improvements in exercise function, behavior, self-esteem and emotional state 6 months after their program ended.
  • Impact of Cardiac Rehabilitation on the Exercise Function of Children with Serious Congenital Heart Disease. J. Rhodes, Pediatrics 2005; 116(6):1339-1345.
    Children with congenital heart disease (CHD) often can’t exercise as well as their peers. Part of this may be due to their heart defect and part may be that they may not be very physically active. A 12-week cardiac rehabilitation program was designed to see if children could improve their ability to exercise safely. Sixteen patients completed the program. Improvements were found in 15 of 16 patients and no patient had any heart problems from the exercise.

Fontan Follow-Up Study

Fontan 3

Fontan 3


FUEL Publications

  • Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial. D.J. Goldberg, Am Heart J. 2018 Jul; 201:1-8.
    This manuscript describes the design of the randomized controlled trial of udenafil in children and adolescents with single ventricle heart disease who have undergone the Fontan operation. This study is powered to detect changes in exercise capacity, ventricular performance, and vascular function. These outcomes are important long-term determinants of health, and changes in these outcomes will be important in understanding whether udenafil has a role as a maintenance medication for this population. The FUEL trial is the largest multi-center randomized, controlled medication trial in congenital heart disease to date. This trial will help to determine whether routine udenafil use is associated with improvement in outcomes associated with long-term health in children and adolescents with single ventricle heart disease who have undergone the Fontan operation. Thirty centers from North America and Korea are participating in the FUEL trial.

Kawasaki Disease Study (Medication)

Kawasaki Disease (KD) Publications

Marfan Trial

Marfan Publications

  • Predictors of Rapid Aortic Root Dilation and Referral for Aortic Surgery in Marfan Syndrome. A. Hoskoppal, Pediatr Cardiol 2018 Jun 11. doi: 10.1007/s00246-018-1916-6.
    This was a secondary analysis of the PHN randomized clinical trial of atenolol vs. losartan in patients with Marfan syndrome that attempted to identify factors that predict rapid aortic root dilation and referral for aortic surgery. We found some statistically significant but only weakly predictive associations between more rapid aortic root dilation and older age, non-white race, and one aortic measurement: higher sinotubular junction z-score. We found similarly statistically significant but only weakly predictive associations between referral for aortic root surgery and some aortic measurements (larger aortic root diameter, higher ascending aorta z-score, and higher ratio between the diameters of the sinotubular junction and ascending aorta). Future studies may clarify whether monitoring generalized proximal aortic dilation and effacement of the sinotubular junction will help to risk-stratify young patients with Marfan syndrome and inform medical and surgical management.
  • Influence of Aortic Stiffness on Aortic-Root Growth Rate and Outcome in Patients With the Marfan Syndrome. ES. Selamet Tierney, Am J Cardiol 2018 May 1;121(9):1094-1101.
    The PHN randomized trial of atenolol versus losartan in Marfan Syndrome showed no treatment differences in the rates of aortic-root growth or clinical outcomes. This analysis examined treatment effects on aortic stiffness and determined whether baseline aortic stiffness predicts aortic-root growth and clinical outcomes. There was no treatment effect on the rate of change for ascending-aorta stiffness index, aortic-root elastic modulus, or ascending-aorta elastic modulus. We found that atenolol therapy was associated with a fall in aortic-root stiffness index, while losartan therapy was not. Higher baseline aortic-root stiffness index and elastic modulus were associated with a smaller decrease in aortic root z-score and increased risk for clinical outcomes. These data suggest that non-invasive aortic stiffness measures may identify patients at higher risk for progressive aortic enlargement and adverse clinical outcomes, potentially allowing for closer monitoring and more aggressive therapy for such patients.
  • Atenolol versus Losartan in Children and Young Adults with Marfan's Syndrome. R. Lacro, N Engl J Med 2014; 371:2061-2071.
  • Characteristics of children and young adults with Marfan syndrome and aortic root dilation in a randomized trial comparing atenolol and losartan therapy. R. Lacro, Am Heart J 2013; 165:828-835.e3.
  • Echocardiographic Methods, Quality Review, and Measurement Accuracy in a Randomized Multicenter Clinical Trial of Marfan Syndrome. E. Selamet Tierney, J Am Soc Echocardiogr 2013; 26(6):657-666.
  • Rationale and Design of a Randomized Clinical Trial of B-Blocker Therapy (Atenolol) Versus Angiotensin II Receptor Blocker Therapy (Losartan) in Individuals with Marfan Syndrome. R. Lacro, Am Heart J 2007; 154:624-631.
    The leading cause of death in people with Marfan syndrome is dilation, tearing and rupture of the aorta, the large artery that delivers blood from the heart to the body. Recent studies in mice with Marfan syndrome showed that treatment with Losartan, a medication used to treat high blood pressure, prevented dilation and improved the structure of the wall of the aorta. This article describes the design of a trial to compare Losartan to the standard medication (Atenolol) for patients with Marfan syndrome. The goal of the trial is to follow over 600 children and young adults for 3 years to determine which drug does a better job slowing the rate of growth of the aorta.

Mitral Regurgitation Study (Observational)

Mitral Regurgitation (ACE-I in MR) Publications

Multi Study (ISV-SVR-SVR II)

Multi Study (ISV-SVR-SVR II) Publications

Normal Electrocardiogram (ECG) Project

Normal Electrocardiogram (ECG) Project

  • Electrocardiograms in Healthy North American Children in the Digital Age. E.V. Saarel, Circ Arrhythm Electrophysiol 2018 Jul;11(7):e005808.
    Electrocardiography is a cornerstone in the cardiac evaluation of children. Wide variation in previously published data, much of which was obtained before the digital era, provided a strong motivation to obtain more reliable data on electrocardiogram (ECG) measurements in healthy children from North America. This study found that most ECG measurements varied by sex and race and differed from prior studies done in smaller, more racially and ethnically homogeneous populations. This study provides valuable data that can be used clinically for interpreting pediatric ECGs in the modern era for diagnosis or screening of heart disease in North America, particularly for Long QT syndrome and left ventricular hypertrophy.

PHN Scholars

PHN Scholars Publications

  • The Pediatric Heart Network Scholar Award programme: a unique mentored award embedded within a multicentre network. L.L. Minich, Cardiol Young 2018 Jun;28(6):854-861.
    The Pediatric Heart Network designed a career development award to train the next generation of clinician-scientists in pediatric cardiology-related research, a historically underfunded area. This manuscript described strengths and weaknesses of the program and the scholars’ academic achievements for the first two funding cycles. Strengths included clarity and fairness of the review, but feedback on the application was not considered useful. The highest rated benefits were expanding the scholar’s collaborative network and increasing publication potential. The 13 scholars were first/senior authors for 97 abstracts and 109 manuscripts, served on 22 PHN committees, and were awarded $9,673,660 in subsequent extramural funding for a return of ~$10 for every Scholar dollar spent.

Residual Lesion Score (RLS)

Residual Lesion Score (RLS) Publications

Single Ventricle Study (Medication)

Single Ventricle (ISV) Publications

Single Ventricle Study (Surgical)

Single Ventricle (SVR) Publications

Single Ventricle Reconstruction Extension Study

Single Ventricle Reconstruction Extension Study

Team 4 Growth