The ACTION VAD registry: A collective five-year experience.

Background: The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) began in 2018 as a collaborative learning health system committed to improving outcomes in pediatric heart failure, including children and adults with congenital heart disease, supported with ventricular assist devices (VADs). This report describes patient and device characteristics, and outcomes through 1-year post-implant.

Methods: The ACTION VAD registry report was created from data submitted to the ACTION learning network from April 2018 to June 2023.

Impact of Heart Transplant Allocation Changes on Waitlist Mortality and Clinical Practice in Pediatric and Adult Patients With Congenital Heart Disease and Cardiomyopathy.

Background: The United Network of Organ Sharing made changes to the priority for allocation of hearts for transplantation (HT) in 2016 for pediatric patients and 2018 for adult patients. Although recent work has evaluated the impact of the revised allocation systems on mechanical circulatory support practices and waitlist outcomes, there are limited data that focus more specifically on the impact of the allocation changes on patients with congenital heart disease (CHD) or cardiomyopathy and how these relationships might differ in pediatric and adult patients.

Methods: The United Network of Organ Sharing database was queried for pediatric (<18 years of age) and adult (18-50 years of age) patients with a CHD or cardiomyopathy diagnosis listed for HT.

Response of health-related quality of life following pediatric/congenital cardiac catheterization procedures.

Background: Health related quality of life (HRQOL) is a patient-reported metric (PRM) that provides a holistic measure of health that is not addressed in traditional outcome measures. The acute responsiveness of HRQOL after pediatric/congenital cardiac catheterization procedures has not, to our knowledge, been studied.

Methods: A single-center prospective cohort study was performed, longitudinally evaluating HRQOL and other PRM in school-age children and adolescents (ages 8-18) undergoing diagnostic and interventional cardiac catheterization procedures prior to their scheduled procedure, and then 1 day, ∼1 month, and ∼3 months after the procedure.

Safety and Efficacy of GLP-1 Agonists in a Cohort of Patients with Fontan Circulation.

There are a growing number of adult patients palliated to a Fontan circulation. As these patients age, many develop symptomatic heart failure (d'Udekem et al in Circulation 130:S32-S38, 2014) that is exacerbated by acquired comorbidities such as obesity and hypertension. Increased body mass index (BMI) and adiposity have been associated with worse hemodynamics and clinical outcomes in these patients (Yogeswaran et al in J Am Hear Assoc: Cardiovasc Cerebrovasc Dis 12:e026732, 2023).

Prevalence and Impact of Recurrent Rejection on Pediatric Heart Transplant Recipients: A PHTS Multi-Institutional Analysis.

Background: Studies evaluating the prevalence and impact of recurrent rejection (RR) in pediatric heart transplant (HT) are sparse.

Objectives: The purpose of this study was to describe prevalence and impact of RR on cardiac allograft vasculopathy (CAV) and graft loss after pediatric HT.

Methods: Data on HT from January 1, 2000, to June 30, 2020, in the Pediatric Heart Transplant Society database were included.

Advances in Topical Botulinum Therapy.

Background: A successfully developed topical botulinum toxin product would overcome the disadvantages of injectable administration commonly seen with all available botulinum products, including the potential pain, bleeding, and bruising associated with injection.

Objective: Identify the disadvantages of injectable administration of botulinum products and advantages of topical administration. Identify previous attempts to develop topical products and review the only topical product candidate (ET-01, abbreviated EMX) under clinical development.

Prolonged Inotrope Use After Surgery for Congenital Heart Disease: A Common Occurrence with a High Burden of Mortality.

Outcomes in patients requiring prolonged inotropes (PI) following surgery for congenital heart disease (CHD) have not been well studied. We aimed to describe the burden of PI use in the immediate postoperative period after CHD surgery and identify risk factors for in-hospital mortality. We conducted a retrospective cohort study using the Pediatric Health Information System® (PHIS) database.

Mechanical Circulatory Support in Pediatric Myocarditis: Support Strategies and Outcomes in a Nationally Representative Cohort.

Background: Myocarditis is a common cause of pediatric heart failure which may require mechanical circulatory support (MCS). The purpose of this study is to describe MCS strategies used in a nationwide cohort of pediatric patients with myocarditis, identify trends over time, and compare outcomes between MCS strategies.

Methods: This study utilized the Kids' Inpatient Database (KID), a national sample of administrative discharge data.

Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States.

Background And Objectives: Standards of care exist to optimize outcomes in Duchenne and Becker muscular dystrophy (DBMD), caused by alterations in the DMD gene; however, there are limited data regarding health care access in these patients. This study aims to characterize outpatient subspecialty care utilization in pediatric patients with DBMD.

Methods: This retrospective cohort study used administrative claims data from IBM MarketScan Medicaid and Commercial Claims and Encounters Research Databases (2013-2018).

Hybrid stage 1 palliation with simultaneous off-pump ventricular assist device placement in neonates with high-risk single ventricle anatomy: Initial experience.

Background: Infants with single ventricle heart disease and severe atrioventricular valve regurgitation have poor outcomes following conventional staged palliation. As such, ventricular assist device (VAD) placement along with hybrid stage 1 palliation has been proposed as a bridge to heart transplant. We present a novel surgical technique for VAD implantation concurrent with hybrid stage 1 that avoids cardiopulmonary bypass.

Cardiopulmonary exercise testing on adaptive equipment in children and adults with Friedreich ataxia.

Introduction/aims: Traditional exercise is often difficult for individuals with Friedreich ataxia (FRDA), and evidence is limited regarding how to measure exercise performance in this population. We evaluated the feasibility, reliability, and natural history of adaptive cardiopulmonary exercise test (CPET) performance in children and adults with FRDA.

Methods: Participants underwent CPET on either an arm cycle ergometer (ACE) or recumbent leg cycle ergometer (RLCE) at up to four visits (baseline, 2 weeks, 4 weeks, and 1 year).

Introduction of transcatheter edge-to-edge repair in patients with congenital heart disease at a children's hospital.

Background: Atrioventricular valve regurgitation (AVVR) is a devastating complication in children and young adults with congenital heart disease (CHD), particularly in patients with single ventricle physiology. Transcatheter edge-to-edge repair (TEER) is a rapidly expanding, minimally invasive option for the treatment of AVVR in adults that avoids the morbidity and mortality associated with open heart surgery. However, application of TEER in in CHD and in children is quite novel.

The Effect of Udenafil on Heart Rate and Blood Pressure in Adolescents With the Fontan Circulation.

The Fontan Udenafil Exercise Longitudinal (FUEL) trial showed that treatment with udenafil was associated with improved exercise performance at the ventilatory anaerobic threshold in children with Fontan physiology. However, it is not known how the initiation of phosphodiesterase 5 inhibitor therapy affects heart rate and blood pressure in this population. These data may help inform patient selection and monitoring after the initiation of udenafil therapy.

Hypertension: An Important But Reversible Cause of Systolic Dysfunction in a Cohort of Pediatric Patients.

Cardiac dysfunction due to hypertension (CDHTN) in pediatrics is not well described. We aimed to describe the presentation and outcomes of pediatric CDHTN and identify clinical features associated with resolution of dysfunction. A single-center retrospective cohort study of patients ≤ 21 years with CDHTN from January 2005-September 2020 was performed.

School age and adolescent heart failure following the Norwood procedure.

Background: Heart failure results in significant morbidity and mortality for young children with hypoplastic left heart syndrome (HLHS) following the Norwood procedure. The trajectory in later childhood is not well described.

Methods: We studied the outcome into adolescence of participants enrolled in the Single Ventricle Reconstruction trial who underwent the Fontan procedure or survived to 6 years without having undergone Fontan procedure.

Reduced incidence of cardiac rejection in multi-organ transplants: A propensity matched study.

Background: Rejection remains a primary cause of graft loss after heart transplant (HT). Recognizing the immunomodulation of multi-organ transplant can enhance our understanding of the mechanisms of cardiac rejection.

Methods: This retrospective cohort study identified patients from the UNOS database with isolated heart (H, N = 37 433), heart-kidney (HKi, N = 1516), heart-liver (HLi, N = 286), and heart-lung (HLu, N = 408) transplants from 2004 to 2019.

Temporal trends in primary payers in pediatric heart transplant and association with long-term survival.

Background: Pediatric heart transplantation (HT) is resource intensive. In adults, there has been an increase in the proportion of HTs funded by public insurance, with post-HT outcomes inferior to those funded by private sources. Trends in the funding of pediatric HT and outcomes in children have not been described.

Cost-effectiveness of bivalirudin in pediatric ventricular assist devices.

Background: Despite recent data suggesting improved outcomes with bivalirudin vs heparin in pediatric Ventricular assist devices (VAD), higher costs remain a barrier. This study quantified trends in bivalirudin use and compared outcomes, resource utilization, and cost-effectiveness associated with bivalirudin vs heparin.

Methods: Children age 0 to 6 year who received VAD from 2009 to 2021 were identified in Pediatric Health Information System.

Cardiopulmonary Exercise Testing in Pediatric Patients With Hypertrophic Cardiomyopathy.

Background: Exercise stress testing (EST) in pediatric hypertrophic cardiomyopathy (HCM) patients has not well described in a large heterogenous cohort.

Objectives: The objective of the study was to determine the clinical utility of EST in pediatric HCM.

Methods: This was a retrospective single-center analysis of HCM patients younger than 21 years who had EST between January 1, 2000, and January 1, 2019.