Cardiac Remodeling in Subclinical Hypertrophic Cardiomyopathy: The VANISH Randomized Clinical Trial.

Importance: Valsartan has shown promise in attenuating cardiac remodeling in patients with early-stage sarcomeric hypertrophic cardiomyopathy (HCM). Genetic testing can identify individuals at risk of HCM in a subclinical stage who could benefit from therapies that prevent disease progression.

Objective: To explore the potential for valsartan to modify disease development, and to characterize short-term phenotypic progression in subclinical HCM.

Delphi Panel Consensus Recommendations for Screening and Managing Childhood Cancer Survivors at Risk for Cardiomyopathy.

Background: Cardiomyopathy is a leading cause of late morbidity and mortality in childhood cancer survivors (CCS). Evidence-based guidelines recommend risk-stratified screening for cardiomyopathy, but the management approach for abnormalities detected when screening asymptomatic young adult CCS is poorly defined.

Objectives: The aims of this study were to build upon existing guidelines by describing the expert consensus-based cardiomyopathy screening practices, management approach, and clinical rationale for the management of young adult CCS with screening-detected abnormalities and to identify areas of controversy in practice.

Valsartan in early-stage hypertrophic cardiomyopathy: a randomized phase 2 trial.

Hypertrophic cardiomyopathy (HCM) is often caused by pathogenic variants in sarcomeric genes and characterized by left ventricular (LV) hypertrophy, myocardial fibrosis and increased risk of heart failure and arrhythmias. There are no existing therapies to modify disease progression. In this study, we conducted a multi-center, double-blind, placebo-controlled phase 2 clinical trial to assess the safety and efficacy of the angiotensin II receptor blocker valsartan in attenuating disease evolution in early HCM.

Exercise Capacity and Predictors of Performance After Fontan: Results from the Pediatric Heart Network Fontan 3 Study.

Impaired exercise following Fontan is a surrogate of morbidity. Single-center longitudinal data exist, but there is a lack of contemporary multi-center data. Ramp cycle ergometry was re-performed in consented participants who had originally participated in the Pediatric Heart Network's Fontan cross-sectional study.

Value of Exercise Stress Echocardiography in Children with Hypertrophic Cardiomyopathy.

Background: Exercise stress echocardiography (ESE) is a valuable diagnostic and prognostic tool in adults with hypertrophic cardiomyopathy (HCM). Inducible and resting left ventricular outflow tract gradients are important predictors of heart failure. However, there are minimal data on the utility of this modality in children.

Baseline Characteristics of the VANISH Cohort.

Background: The VANISH trial (Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hypertrophic Cardiomyopathy) targeted young sarcomeric gene mutation carriers with early-stage hypertrophic cardiomyopathy (HCM) to test whether valsartan can modify disease progression. We describe the baseline characteristics of the VANISH cohort and compare to previous trials evaluating angiotensin receptor blockers.

Methods: Applying a randomized, double-blinded, placebo-controlled design, 178 participants with nonobstructive HCM (age, 23.

Comparison of echocardiographic measurements to invasive measurements of diastolic function in infants with single ventricle physiology: a report from the Pediatric Heart Network Infant Single Ventricle Trial.

Background: While echocardiographic parameters are used to quantify ventricular function in infants with single ventricle physiology, there are few data comparing these to invasive measurements. This study correlates echocardiographic measures of diastolic function with ventricular end-diastolic pressure in infants with single ventricle physiology prior to superior cavopulmonary anastomosis.

Methods: Data from 173 patients enrolled in the Pediatric Heart Network Infant Single Ventricle enalapril trial were analysed.

Regional practice norms for the care of childhood cancer survivors at risk for cardiomyopathy: A Delphi study.

Background: Treatment-associated cardiomyopathy is a leading cause of morbidity and mortality for childhood cancer survivors (CCS). As evidence is not available to guide the management of CCS at risk for cardiomyopathy, we aim to describe the collective opinion of regional experts for the care of these patients using a consensus-based Delphi methodology.

Procedure: Nineteen physicians from the New England region who care for CCS treated with cardiotoxic therapy (anthracyclines, thoracic radiation) participated in a Delphi panel querying their management approach, using three rounds of anonymous questionnaires formatted as five clinical scenarios.

Markers of Bone Mineral Metabolism and Cardiac Structure and Function in Perinatally HIV-Infected and HIV-Exposed but Uninfected Children and Adolescents.

Background: Disordered bone mineral metabolism and low vitamin D concentrations are associated with cardiovascular abnormalities; few studies have evaluated this relationship in HIV-infected youth.

Setting: The Adolescent Master Protocol is a Pediatric HIV/AIDS Cohort Study network study conducted across 14 US sites.

Methods: Among perinatally HIV-infected (PHIV) and perinatally HIV-exposed but uninfected (PHEU) youth enrolled in the Adolescent Master Protocol, we evaluated associations of vitamin D [measured as 25-hydroxy-vitamin D (25-OHD)], parathyroid hormone (PTH), calcium, phosphate, and fibroblast growth factor-23 (FGF-23) concentrations with echocardiographic measures of left ventricular (LV) structure, function, and concentrations of NT-proBNP, a biomarker of cardiac damage.

Cardiac and inflammatory biomarkers in perinatally HIV-infected and HIV-exposed uninfected children.

Objectives: To compare distributions of serum cardiac and inflammatory biomarkers between perinatally HIV-infected (PHIV) and perinatally HIV-exposed uninfected (PHEU) children, to evaluate their associations with echocardiographic measures, and among PHIV youth, with antiretroviral therapy (ART) and HIV disease severity measures.

Design: Cross-sectional analysis of temporally paired serum samples for biomarkers and echocardiograms in a prospective multicenter cohort study of PHIV and PHEU youth.

Methods: Serum samples were analyzed among 402 youth in the PHACS Adolescent Master Protocol (AMP) for high-sensitivity cardiac troponin-T (hs-cTnT, a cardiomyocyte injury marker), N-terminal-pro-brain natriuretic peptide (NT-proBNP, a myocardial stress marker), and inflammatory markers [high-sensitivity C-reactive protein, interleukin (IL)-1, IL-6, IL-8, IL-10, IL-18, tumor necrosis factor-α (TNF-α), and soluble TNF receptor II (sTNF-RII)].

Longitudinal Outcomes of Patients With Single Ventricle After the Fontan Procedure.

Background: Multicenter longitudinal objective data for survival into adulthood of patients who have undergone Fontan procedures are lacking.

Objectives: This study sought to describe transplant-free survival and explore relationships between laboratory measures of ventricular performance and functional status over time.

Methods: Exercise testing, echocardiography, B-type natriuretic peptide, functional health assessment, and medical history abstraction were repeated 9.

The Effect of the Superior Cavopulmonary Anastomosis on Ventricular Remodeling in Infants with Single Ventricle.

Background: Infants with single ventricular physiology have volume and pressure overload that adversely affect ventricular mechanics. The impact of superior cavopulmonary anastomosis (SCPA) on single left ventricles versus single right ventricles is not known.

Methods: As part of the Pediatric Heart Network placebo-controlled trial of enalapril in infants with single ventricular physiology, echocardiograms were obtained before SCPA and at 14 months and analyzed in a core laboratory.

Assessment of Diastolic Function in Single-Ventricle Patients After the Fontan Procedure.

Background: Patients with functional single ventricles after the Fontan procedure have abnormal cardiac mechanics. The aims of this study were to determine factors that influence diastolic function and to describe associations of diastolic function with current clinical status.

Methods: Echocardiograms were obtained as part of the Pediatric Heart Network Fontan Cross-Sectional Study.

Systolic-diastolic functional coupling in healthy children and in those with dilated cardiomyopathy.

Systolic and diastolic function affect dilated cardiomyopathy (DCM) outcomes. However, systolic-diastolic coupling, as a distinct characteristic, may itself affect function but is poorly characterized. We hypothesized that echocardiographic left ventricular (LV) longitudinal systolic tissue velocities (S') correlate with diastolic longitudinal velocities (E') and that their relationship is associated with ventricular function and that this relationship is impaired in pediatric DCM.

Assessment of Quality of Life in Young Patients with Single Ventricle after the Fontan Operation.

Objectives: To assess self-reported quality of life (QOL) in a large multicenter cohort of adolescent and young adults surviving Fontan.

Study Design: Cross-sectional. The Pediatric Quality of Life Inventory (PedsQL) was administered to 408 survivors of Fontan ages 13-25 years enrolled in the Pediatric Heart Network Fontan Follow-up Study.

The reproducibility and absolute values of echocardiographic measurements of left ventricular size and function in children are algorithm dependent.

Background: Several quantification algorithms for measuring left ventricular (LV) size and function are used in clinical and research settings. The aims of this study were to investigate the effects of measurement algorithm and beat averaging on the reproducibility of measurements of the left ventricle and to assess the magnitude of agreement among the algorithms in children with dilated cardiomyopathy.

Methods: Echocardiograms were obtained in 169 children from eight clinical centers.

Survival data and predictors of functional outcome an average of 15 years after the Fontan procedure: the pediatric heart network Fontan cohort.

Objective: Multicenter longitudinal outcome data for Fontan patients surviving into adulthood are lacking. The aim of this study was to better understand contemporary outcomes in Fontan survivors by collecting follow-up data in a previously well-characterized cohort.

Design: Baseline data from the Fontan Cross-Sectional Study (Fontan 1) were previously obtained in 546 Fontan survivors aged 11.

Recovery of echocardiographic function in children with idiopathic dilated cardiomyopathy: results from the pediatric cardiomyopathy registry.

Objectives: This study sought to determine the incidence and predictors of recovery of normal echocardiographic function among children with idiopathic dilated cardiomyopathy (DCM).

Background: Most children with idiopathic DCM have poor outcomes; however, some improve.

Methods: We studied children <18 years of age from the Pediatric Cardiomyopathy Registry who had both depressed left ventricular (LV) function (fractional shortening or ejection fraction z-score <-2) and LV dilation (end-diastolic dimension [LVEDD] z-score >2) at diagnosis and who had at least 1 follow-up echocardiogram 30 days to 2 years from the initial echocardiogram.

Factors associated with serum B-type natriuretic peptide in infants with single ventricles.

Data regarding the value of B-type natriuretic peptide (BNP) measurements in infants with a single-ventricle (SV) physiology are lacking. This analysis aimed to describe the BNP level changes in infants with an SV physiology before and after superior cavopulmonary connection (SCPC) surgery. Levels of BNP were measured by a core laboratory before SCPC (at 5.

Variability of M-mode versus two-dimensional echocardiography measurements in children with dilated cardiomyopathy.

M-mode and 2-dimensional (2D) echocardiographic imaging are routinely used to quantify left-ventricular (LV) size and function in pediatric patients with dilated cardiomyopathy (DCM). The reproducibility of and correlation between these techniques are unknown. This analysis sought to compare interreader, intrareader, and interacquisition reproducibility of M-mode versus 2D measurements in pediatric DCM patients.

Predictors of disease progression in pediatric dilated cardiomyopathy.

Background: Despite medical advances, children with dilated cardiomyopathy (DCM) remain at high risk of death or need for cardiac transplantation. We sought to identify predictors of disease progression in pediatric DCM.

Methods And Results: The Pediatric Heart Network evaluated chronic DCM patients with prospective echocardiographic and clinical data collection during an 18-month follow-up.

Functional health status in children and adolescents after Fontan: comparison of generic and disease-specific assessments.

Purpose: The aim of this study was to compare associations between generic versus disease-specific functional health status assessments and patient and clinical characteristics for patients with severe congenital heart disease.

Methods: This was a cross-sectional observational study involving 325 single ventricle patients, aged 10-18 years, after Fontan procedure. Enrolled patients underwent a medical history review, laboratory testing, and assessment of the functional health status by completion of the generic Child Report Child Health Questionnaire and the disease-specific Congenital Heart Adolescent and Teenage questionnaire.

Factors impacting echocardiographic imaging after the Fontan procedure: a report from the pediatric heart network fontan cross-sectional study.

Echocardiographic image quality in Fontan survivors may be limited by a variety of factors. We sought to describe echocardiographic quality and factors associated with study quality in subjects participating in the Pediatric Heart Network Fontan Cross-Sectional Study. Echocardiograms were obtained at 7 clinical sites using a standard protocol.

Ventricular mechanics in patients with aortic valve disease: longitudinal, radial, and circumferential components.

Background: Reduced long-axis shortening despite enhanced global function has been reported in aortic stenosis. We sought to improve the understanding of this phenomenon using multi-dimensional strain analysis in conjunction with the evaluation of left ventricular rotation and twist - ventricular torsion - using tissue Doppler techniques.

Methods: A total of 57 patients with variable severity of aortic stenosis, aortic regurgitation, or mixed aortic valve disease, subdivided into six groups, were studied.

Superior cavopulmonary anastomosis timing and outcomes in infants with single ventricle.

Objectives: We sought to identify factors associated with the timing and surgical outcomes of the superior cavopulmonary anastomosis.

Methods: The Pediatric Heart Network's Infant Single Ventricle trial database identified participants who underwent superior cavopulmonary anastomosis. Factors potentially associated with age at superior cavopulmonary anastomosis, length of stay and death by 14 months of age were evaluated.