BACKGROUND:Long-term outcomes after Fontan vary widely. Although pre-Fontan hemodynamics predict early failure, their association with long-term outcomes remains unclear. We hypothesized that pre-Fontan hemodynamics predict long-term risk of death or transplantation. METHODS:We analyzed data from the Pediatric Cardiac Care Consortium, a US-based multicenter registry including patients undergoing first-time Fontan with pre-Fontan catheterization and long-term follow-up. Patients undergoing a Fontan procedure before 18 years of age at any time between 1982 and 2011 were included in the study. Outcomes of interest were in-hospital Fontan failure (death or takedown) and postdischarge death or transplantation, identified through matching with the National Death Index and the Organ Procurement and Transplantation Network through 2022. Associations between pre-Fontan hemodynamics and long-term risk for death or transplantation were assessed with Kaplan-Meier survival curves and extended Cox regression. RESULTS:Among 1175 patients (736 [62.6%] male, 626 [53.3%] with systemic left ventricle), 1111 were discharged with Fontan physiology. Over a median postdischarge follow-up of 20.6 years (interquartile range, 18.2-24.4 years), 85 deaths and 49 transplantations occurred. Pre-Fontan mean pulmonary arterial pressure was the strongest hemodynamic predictor of postdischarge outcomes with a continuous association and no clear inflection point; 25-year transplantation-free survival declined from 83.7% (95% CI, 77.6-88.3) in the low mean pulmonary arterial pressure tertile to 73.7% (95% CI, 65.5-80.3) in the highest tertile (log-rank P=0.02). Each 1-SD increase in mean pulmonary arterial pressure was associated with 1.33-fold higher odds of in-hospital failure (adjusted odds ratio, 1.33 [95% CI, 1.00-1.77]; P=0.05) and a 2.2-fold higher hazard of death or transplantation (adjusted hazard ratio, 2.20 [95% CI, 1.62-3.00]; P<0.01) estimated at discharge. This hazard declined 3% per year after discharge (adjusted hazard ratio per year, 0.97 [95% CI, 0.95-0.99]; P<0.01) and resolved by 17 years in patients with systemic right ventricle and by 23 years in those with systemic left ventricle. Additional independent risk factors included systemic right ventricle versus systemic left ventricle (adjusted hazard ratio, 2.39 [95% CI, 1.65-3.46]; P<0.01) and delayed Fontan completion (>4 years of age versus 2 to 4 years of age; adjusted hazard ratio, 1.80 [95% CI, 1.25-2.60]; P=0.02). CONCLUSIONS:Elevated pre-Fontan mean pulmonary arterial pressure is a strong predictor of in-hospital and long-term post-Fontan risk of death or transplantation. Systemic right ventricle and delayed Fontan completion (>4 years of age) further increased risk. These findings support early Fontan consideration and ongoing hemodynamic surveillance to optimize long-term outcomes.

Background: Galectin-3 and soluble suppression of tumorigenicity-2 (sST2) are biomarkers of fibrosis and inflammation, with prognostic value in adult cardiac failure both with and without mechanical circulatory support. Their utility in pediatric venoarterial extracorporeal membrane oxygenation (VA-ECMO), especially across different age, remains unclear. Research Question: Can early Galectin-3 and sST2 levels, with or without age context, predict mortality in pediatric patients with cardiac failure requiring VA-ECMO? Methods: We prospectively enrolled 34 pediatric patients on VA-ECMO. Plasma Galectin-3 and sST2 were measured from serial blood samples collected pre-cannulation, at 2-, 4-, and 6-hours post-cannulation, daily up to day 8, and at decannulation and the following day. Levels were quantified using ELISA. Biomarker trends were analyzed, using peak values from the first 3 days to compare outcomes between survivors (discharged) and non-survivors. Logistic regression models assessed the predictive performance of Galectin-3 and sST2 individually and in combination with age. Discrimination was evaluated using ROC curves and Youden’s Index. Results: The cohort included 11 neonates (32%), 16 infants (47%), 5 children (15%), and 2 adolescents (6%). Thirteen patients (38%) were male. Diagnoses included extracorporeal cardiopulmonary resuscitation (38%), failure to wean from cardiopulmonary bypass (26%), cardiomyopathy/myocarditis (24%), and cardiac failure not otherwise specified (15%). Fifteen patients survived to discharge. The median duration of VA-ECMO support was longer in non-survivors (8 days [IQR 5–10]) compared to survivors (5 days [IQR 4–6.5]). Mean Galectin-3 levels were elevated early and declined over time but remained persistently higher in non-survivors. Mean sST2 peaked within 48 hours and declined in both groups, with a longer elevation in non-survivors. Individually, neither biomarker was predictive (AUC = 0.55 each). However, combining both with age improved discrimination (AUC = 0.73, p = 0.02). Optimal thresholds were identified: Galectin-3 ≥ 21.1 ng/mL and sST2 ≥ 1820.6 ng/mL. Conclusion: Early serial sampling of Galectin-3 and sST2 revealed outcome-related temporal patterns. While neither marker alone was predictive, their combination with patient age improved prognostic model in younger patients. These findings support age-adjusted biomarker use for risk stratification in pediatric ECMO. Validation in larger cohorts is warranted.

Introduction: Lamin A/C ( LMNA ) gene mutations result in a heterogeneous group of inherited conditions termed laminopathies. Cardiac manifestations of laminopathies include atrial and ventricular arrhythmias, atrioventricular conduction disorders, and cardiomyopathy, with or without skeletal muscle involvement. Because this disease is rare and typically presents in adulthood, pediatric data is limited. Methods: We identified patients < 18 years with genetically confirmed mutations in LMNA followed at our center between 2003 and 2021. Clinical phenotypes along with cardiac test results were retrospectively catalogued. Results: We identified 12 patients with a median age of 5 years at diagnosis (IQR 4-11 years) with a LMNA mutation. Nine (75%) patients were male and 10 (83%) had associated skeletal muscle involvement. Of the 12 patients, 10 (83%) developed cardiac manifestations over a median follow up period of 111 months (IQR 91-116 months). Seven patients (58%) had documented arrhythmias (AV block, ventricular/atrial tachycardias) and 3 (25%) underwent pacemaker and/or implantable cardiac defibrillator placement. Three (25%) patients were diagnosed with cardiomyopathy with 1 (8%) requiring a ventricular assist device and 1 (8%) requiring continuous intravenous inotropes and subsequent transplant. Three (25%) patients died during the study period, one awaiting transplant, one from complications post-transplant, and one from non-cardiac causes. Conclusions: Cardiac involvement, specifically cardiomyopathy and progressive conduction system abnormalities, were common in pediatric patients with LMNA mutations. Early genetic diagnosis of laminopathies followed by frequent surveillance for dysrhythmia and dysfunction is necessary for more timely initiation of advanced cardiac therapies to prevent adverse events. More comprehensive phenotype-genotype correlation is strongly needed.

Introduction/Background: Congenital heart disease (CHD) is the leading cause of birth defects in the United States and contributes to billions of dollars in healthcare resource utilization each year. Rurality, as a social determinant of health, can exacerbate disparities in access to care, negatively impact the rate of prenatal detection of CHD, and increase healthcare resource utilization. Research Question/Hypothesis: We hypothesized that patients living in rural areas would have lower rates of outpatient (OP) follow-up and higher rates of emergency department (ED) and inpatient (IP) hospital stays relative to patients living in urban or suburban areas. Methods/Approach: A retrospective cohort study of pediatric (0-17 years) CHD patients with an index visit at Nationwide Children’s Hospital (Columbus, OH) from 2013-2023 was conducted. Rurality was determined based on USDA Rural-Urban Commuting Area scores ≥ 4. Patients with at least one year of follow-up were retained for regression analysis of utilization rates. Quasi-Poisson regression models with offset terms were used to elucidate incidence rate ratios (IRR), 95% confidence intervals (CI), and p-values for utilization metrics. The log-rank test was used to compare mortality by rurality. Results/Data: Of the 13,770 patients analyzed, 28.29% (3,896) were from rural areas. Patients from rural areas had an increased median driving distance at 60.3 miles versus 11.5 miles for those from non-rural areas. Rural patients additionally had a lower Child Opportunity Index (COI), as 59.3% (2,312) were considered “Low “or “Very Low” compared to 45.0% (4,447) of their non-rural counterparts. Rural patients had 1.23 (95% CI: 1.16, 1.30; p < 0.001) times the rate of OP cardiology visits, 0.58 (95% CI: 0.53, 0.62; p < 0.001) times the rate of ED visits, and no significant difference in rates of IP admissions (IRR = 1.04; 95% CI: 0.96, 1.13; p = 0.3) or mortality (log-rank p = 0.4) compared to non-rural patients. Conclusions: Rural residency was associated with higher rates of OP cardiology visits, lower rates of ED visits, and similar rates of IP admissions and mortality. Limitations include lack of an all-payers claims database in our state, therefore only local records were utilized in this study. Future research could investigate the utilization of satellite clinics to further categorize OP cardiology visits by location.

The care of the right ventricle (RV) following left ventricular assist device (LVAD) implantation remains a major clinical challenge, with right ventricular failure (RVF) contributing significantly to morbidity and mortality. While much of the literature focuses on preoperative risk stratification and long-term management, there is limited guidance on the immediate postoperative period from a critical care perspective, particularly in pediatric patients. This review aims to provide practical guidance on the bedside management of the RV in the perioperative period following LVAD implantation in children with biventricular circulation, offering a framework for optimizing RV function and preventing failure. We discuss the pathophysiology of RVF in this setting, highlight key hemodynamic principles, and explore targeted interventions including volume management, inotropic and pulmonary vasodilator support, ventilatory strategies, mechanical circulatory support options, and strategies to mitigate secondary organ dysfunction. By addressing these pediatric-specific critical care considerations, we aim to assist bedside providers in optimizing outcomes for children undergoing LVAD implantation.

Recent data demonstrate worse heart transplant (HTx) outcomes in children with shorter VAD durations, but do not account for VAD adverse events (AEs)Es. We compared outcomes of patients bridged to HTx with < 30 vs. ≥ 30 days of VAD support in an earlier era by assessing both VAD and HTx risk factors. We merged data from the PediMACS and Pediatric Heart Transplant Study registries to compare one-year post-HTx mortality in patients with < 30 vs. ≥ 30 days of pre-HTx VAD support between 2012 and 2018. We used inverse probability of treatment weighting using propensity scores (PS) to control for confounders, including age, blood type, allosensitization, cardiac diagnosis (cardiomyopathy, congenital heart disease, or myocarditis), VAD support type (left, right, single, or biventricular VAD), and pre-Tx mechanical ventilation and vasoactive support. Among 271 patients, there were 60 in the < 30-days and 211 in the ≥ 30-days groups. At HTx, the < 30-days group used more ventilation (34

Background: Resilience describes an individual’s ability to leverage internal strengths, external support, and acquired skills to maintain wellbeing amidst stressors. Individuals with Fontan-palliated congenital heart disease (CHD) often face physical and psychological stressors, yet some report resilience amidst these circumstances. While resilience is linked to important physical and psychological outcomes in other chronic illnesses, it is underexplored in pediatric and young adult CHD. Research Question: This prospective study aimed to 1) characterize self-reported resilience in a multi-center sample of children and young adults with Fontan circulatory failure (FCF) and 2) identify demographic, physical, and psychological factors correlated with resilience. Methods: Pediatric and young adult patients with Fontan circulation referred for heart failure evaluation at 20 North American pediatric heart centers were eligible. Participants completed the Connor-Davidson Resilience Scale (CD-RISC©). CD-RISC© scores range from 0-40. In adults, scores < 29 represent the lowest quartile. In pediatric cancer and adult CHD, mean scores of 29 have been reported. Participants also completed a measure of disease burden/benefit and NIH PROMIS surveys assessing life satisfaction, purpose in life, depression, and anxiety. Medical data was obtained from chart reviews, and frailty was assessed using the Lurz-Wilde pediatric frailty criteria. Results: The study included 131 participants (60% male, 78% White, median age 16.1 years) with a mean CD-RISC score of 27.2 (SD=7.2; Figure 1). Lower resilience was associated with younger age (p<0.0001), female sex (p=0.01), and hypoplastic left heart syndrome (HLHS; p=0.049). Lower resilience (p=0.001), higher anxiety (p=0.01), and high self-reported disease burden (p=0.02) correlated with frailty. Few objective FCF comorbidities were associated with resilience. In univariate analysis, protein losing enteropathy (PLE; p=0.04) and clinical cyanosis (p=0.053) were associated with lower resilience. In multivariate analysis including age, sex, HLHS diagnosis, and PLE, only younger age and female sex remained independently associated with lower resilience. Conclusion: Although young people with FCF report high resilience, many indicate opportunities for intervention. The study underlines resilience as a modifiable, critical target for intervention in complex CHD, highlighted by its strong link with frailty over less modifiable comorbidities.

BACKGROUND:A pediatric national heart review board (NHRB) and exception guidance document to standardize decision-making were implemented in 2021 to reduce variability and ensure equity in status exceptions for pediatric candidates. We evaluated the hypothesis that these changes decreased center variability and racial disparities within the granted exceptions. METHODS:Guidance document and pediatric NHRB were operational by February and June 2021, respectively. Candidates were stratified by listing date into: Era 1, pre-policy changes (July 2018-June 2020) and Era 2, post-policy changes (July 2021-June 2023). Mixed effects logistic regression models evaluated individual and center-level predictors of receiving status 1A and 1B exceptions (E) pre- and post-policy implementation. RESULTS:Of 1,275 Era 1 listees, 15% received a 1A(E), with significant center variation. Black listees had lower likelihood of receiving 1A(E) (OR 0.57 [95% CI 0.34-0.94]), controlling for age, diagnosis, and center effects. Among 1,369 Era 2 listees, 14% received status 1A(E). Race was not associated with 1A(E), when controlling for the same variables, and center effect was not significant. While children listed 1B(E) increased from 12% to 16% from Era 1 to Era 2, in both eras, Black children were less likely to receive 1B(E) (OR 0.56 [95% CI 0.33-0.94] in Era 1, and 0.56 [0.34 - 0.91]) in Era 2). Center effect was significant in both eras. CONCLUSIONS:Since implementing exception guidance and a pediatric review board, variation by center and patient race/ethnicity in 1A exceptions has been reduced. Center variation and racial disparities persist among 1B exceptions.

As the number of patients with congenital heart disease (CHD) continues to increase, the burden of heart failure (HF) in this population requires innovative strategies to individualize management. Given the success of implanted invasive hemodynamic monitoring (IHM) with the CardioMEMSTM HF system in adults with acquired HF, this is often suggested for use in patients with CHD, though published data are limited to case reports and case series. Therefore, this review summarizes the available published reports on the use of IHM in patients with complex CHD, describes novel applications, and highlights future directions for study. In patients with CHD, IHM has been used across the lifespan, from age 3 years to adulthood, with minimal device-related complications reported. IHM uses include (1) prevention of HF hospitalizations; (2) reassessment of hemodynamics after titration of medical therapy without repeated cardiac catheterization; (3) serial monitoring of at-risk patients for pulmonary hypertension to optimize timing of heart transplant referral; (4) and hemodynamic assessment with exercise (5) or after ventricular assist device placement. IHM has the potential to reduce the number of cardiac catheterizations in anatomically complex patients and, in patients with Fontan circulation, IHM pressures may have prognostic implications. In conclusion, though further studies are needed, as patients with CHD age and HF is more prevalent, this tool may assist CHD physicians in caring for this complex patient population.

Arrhythmogenic cardiomyopathy (ACM) is an inherited cardiomyopathy associated with fibrofatty tissue replacement of the ventricular tissue. The disease can cause ventricular dysfunction and arrhythmias and can increase the risk of sudden cardiac death. This cardiomyopathy can have variable clinical presentations, especially in the pediatric and young adult populations. In this report, we describe the case of an 18-year-old female with myocarditis as the initial presentation of ACM. She presented following a resuscitated cardiac arrest due to ventricular arrhythmia. On arrival, myocardial edema and delayed gadolinium enhancement were present on cardiac magnetic resonance imaging, with no ventricular changes observed, making the diagnosis consistent with myocarditis. Genetic testing revealed a pathogenic mutation in the desmoplakin gene consistent with ACM. Given the unconventional initial presentation of this patient's disease, early consideration of genetic testing may be beneficial to aid in the early diagnosis and management of ACM in young patients.

Introduction: Infants with hypoplastic left heart syndrome undergoing single ventricle (SV) palliation may need early heart transplant. We aimed to evaluate predictors of transplant referral (TxR) and death without TxR, with focus on neighborhood opportunity and race/ethnicity, to determine if disparities in TxR contribute to increased mortality seen in certain populations. Methods: Retrospective cohort study of all infants undergoing stage 1 (S1) SV palliation enrolled in the National Pediatric Cardiology Quality Improvement Collaborative from 2016-2022 was performed. Competing risk analysis was used to evaluate predictors of TxR and death without TxR through one year. Results: A total of 3099 infants underwent S1 palliation during our study period. Black children had lower median birth weight compared to non-Hispanic white and Hispanic children (p <0.001) as did those from low compared to higher opportunity neighborhoods (p <0.001). There were no other significant pre-operative or S1 procedure differences. At one year, 79% of children were alive, 16% had died, and 5% had a TxR (Figure). In multivariable analysis, risk factors for TxR included hybrid S1 palliation [sHR 2.43 (95% CI 1.54, 3.81], moderate or greater tricuspid regurgitation (TR) [sHR 3.39 (95% CI 1.80 – 6.40)] and female sex [sHR 1.73 (95% CI 1.24 – 2.40)]. Lower birth weight, genetic syndrome, pre-operative TR, hybrid S1 palliation, and small S1 volume (<8 /yr) center were associated with death without TxR. Neither race/ethnicity nor neighborhood opportunity was associated with TxR or death without TxR when controlling for these risk factors. Among those with pre-operative moderate or greater TR, race/ethnicity was associated with TxR in multivariable analysis, with TxR less likely for Black and Other race children compared to white children (p<0.001 for both). Conclusion: One-year survival after S1 palliation without TxR approaches 80%. No clear disparities in death or TxR by race or neighborhood were seen overall, though lower rates of TxR for Black and Other race children with high-risk cardiac features were present. Standardized guidelines for timely TxR may help further reduce death and mitigate potential disparities in this SV population.

PURPOSE:Paediatric patients with heart failure requiring ventricular assist devices are at heightened risk of neurologic injury and psychosocial adjustment challenges, resulting in a need for neurodevelopmental and psychosocial support following device placement. Through a descriptive survey developed in collaboration by the Advanced Cardiac Therapies Improving Outcomes Network and the Cardiac Neurodevelopmental Outcome Collaborative, the present study aimed to characterise current neurodevelopmental and psychosocial care practices for paediatric patients with ventricular assist devices. METHOD:Members of both learning networks developed a 25-item electronic survey assessing neurodevelopmental and psychosocial care practices specific to paediatric ventricular assist device patients. The survey was sent to Advanced Cardiac Therapies Improving Outcomes Network site primary investigators and co-primary investigators via email. RESULTS:Of the 63 eligible sites contacted, responses were received from 24 unique North and South American cardiology centres. Access to neurodevelopmental providers, referral practices, and family neurodevelopmental education varied across sites. Inpatient neurodevelopmental care consults were available at many centres, as were inpatient family support services. Over half of heart centres had outpatient neurodevelopmental testing and individual psychotherapy services available to patients with ventricular assist devices, though few centres had outpatient group psychotherapy (12.5%) or parent support groups (16.7%) available. Barriers to inpatient and outpatient neurodevelopmental care included limited access to neurodevelopmental providers and parent/provider focus on the child's medical status. CONCLUSIONS:Paediatric patients with ventricular assist devices often have access to neurodevelopmental providers in the inpatient setting, though supports vary by centre. Strengthening family neurodevelopmental education, referral processes, and family-centred psychosocial services may improve current neurodevelopmental/psychosocial care for paediatric ventricular assist device patients.

We sought to develop and validate a new risk stratification score for mortality for children supported with a ventricular assist device (VAD). This retrospective, multicenter study used data from patients undergoing VAD implantation between April 2018 and February 2023 at 44 participating institutions in the Advanced Cardiac Therapies Improving Outcomes (ACTION) network. Multivariable Cox proportional-hazards modeled mortality after VAD implantation. A total of 1,022 patients were enrolled. The 1 year mortality was 19% (95% confidence interval [CI]: 16–23). The multivariable model was used to build the ACTION VADs risk stratification score with four components: ventilation, advanced organ support (dialysis or ECMO), diagnosis, and size (weight ≤5 kg). One point is added for each risk factor. Based on the sum of the risk factors, patients were classified into four classes: class 0—green (4% mortality at 1 year), class 1—yellow (16% mortality at 1 year), class 2—orange (21% mortality at 1 year), and class 3 or higher—red (42% mortality at 1 year). The score performed well, with area under the curve (AUC) of 0.72 and excellent calibration. The ACTION VADs score for mortality can be calculated easily and offers risk stratification and prognostic information for pediatric VAD candidates. This is the first validated risk assessment tool for pediatric mechanical circulatory support.

Background Pulmonary hypertension (PH) is a common complication in patients with complete dextro-transposition of the great arteries (TGA) after atrial switch (D-TGA/AS) and congenitally corrected TGA (ccTGA). In this population with subaortic right ventricles (sRVs), echocardiography is a poor screening tool for PH; implantable invasive haemodynamic monitoring (IHM) could be used for this purpose, but data are limited. The aim of this study is to report on novel uses of IHM in patients with sRV. Methods This retrospective study describes the uses of IHM, impact of IHM on heart failure hospitalisation (HFH) and device-related complications in adults with sRV from a single centre (2015–2022). Results IHM was placed in 18 patients with sRV (median age 43 (range 30–54) years, 8 female, 16 with D-TGA/AS, 2 with ccTGA); 16 had moderate or severe sRV systolic dysfunction, 13 had PH on catheterisation. IHM was used for (1) Medical therapy titration, (2) Medical management after ventricular assist device in patients with transplant-limiting PH and (3) Serial monitoring of pulmonary artery pressures without repeat catheterisations to help identify the optimal time for heart transplant referral. In follow-up (median 23 months), HFHs/year were similar to the year prior to IHM (median 0 (IQR 0–1.0) before vs 0 (0–0.8) after, p=0.984). Device migration occurred in one, without long-term sequelae. Conclusions Uses of IHM in patients with sRV are described which may minimise the need for serial catheterisations in a population where PH is prevalent. HFHs were low overall but not impacted by IHM. One device-related complication occurred without long-term consequence.