Susan W. Denfield

Background/Objectives: Pediatric heart failure (HF) is a complex clinical syndrome with diverse etiologies and presentations. Primary care providers (PCPs) are critical in early recognition, family education, and coordination of ongoing care in collaboration with pediatric cardiologists. This review provides a comprehensive guide for PCPs to understand the causes, symptoms, treatments, and longitudinal considerations for children with HF. Methods: We conducted a narrative review synthesizing published evidence and expert consensus guidelines, focusing on the clinical presentation, treatment options, and longitudinal care strategies in children with HF. Results: HF in children most commonly results from congenital heart disease and cardiomyopathy, but includes a wide range of etiologies. Presenting symptoms differ by age, with subtle signs often mistaken for common pediatric illnesses. Treatments range from disease-modifying medications to surgical and transplant therapies. Psychosocial support, mental health, and family-centered care are critical components of long-term management. Conclusions: PCPs are essential partners in the care of children with HF. A proactive, collaborative, and informed approach can improve quality of life, reduce hospitalizations, and support families navigating this challenging diagnosis.

Arrhythmogenic cardiomyopathy (AC) is a disease that involves electromechanical uncoupling of cardiomyocytes. This leads to characteristic histologic changes that ultimately lead to the arrhythmogenic clinical features of the disease. Initially thought to affect the right ventricle predominantly, more recent data show that it can affect both the ventricles or the left ventricle alone. Throughout the recent era, diagnostic modalities and criteria for AC have continued to evolve and our understanding of its clinical features in different age groups as well as the genotype to the phenotype correlations have improved. In this review, we set out to detail the epidemiology, etiologies, presentations, evaluation, and management of AC across the age continuum.

Background: Although ventricular failure is a late finding in adults with AC, we hypothesize that this is a presenting symptom in pediatric heart failure patients who undergo HT and that their ventricular arrhythmia burden could differentiate AC from other cardiomyopathies.Methods: We performed a single‐center retrospective cohort study reviewing 457 consecutive pediatric (≤18 years) HT recipients at our institution. Explanted hearts were examined to establish the primary diagnosis, based on pathologic findings. Demographic and clinical variables were compared between AC versus non‐HCM cardiomyopathy cases.Results: Forty‐five percent (n = 205/457) had non‐HCM cardiomyopathies as the underlying primary diagnosis. Ten cases (10/205 = 4.9%) were diagnosed with AC. All 10 had biventricular disease. In 8/10 patients (80%), AC diagnosis was unrecognized pre‐HT. Compared with non‐AC cardiomyopathies, the AC group was older at diagnosis (9.3 years vs. 4.3 years, p = .012) and transplant (11.1 years vs. 6.5 years, p = .010), had more ventricular arrhythmias (80.0% vs 32.8%, p = .003), and required more anti‐arrhythmic use (80.0% vs 32.3%, p = .001). Genetic testing yielded causative pathogenic variants in all tested individuals (n = 5/5, 100%).Conclusion: AC is often an unrecognized cardiomyopathy pretransplant in children who undergo HT. Pediatric non‐HCM phenotypes with heart failure who have a significant ventricular arrhythmia burden should be investigated for AC.

Background: Guidance and data on ventricular assist device (VAD) support for children with chemotherapy‐induced cardiomyopathy, particularly within the first 2 years after chemotherapy, are limited.Methods: We performed a single‐center retrospective case series, reviewing medical records of children ®. Median age at cancer diagnosis was 6 years (IQR 4.5–10 years). Median dose of anthracycline received was 540 mg/m² (IQR 450–630 mg/m²). All patients developed HF within 1 year after initiation of cancer treatment (median 8 months, IQR 6–11.5 months) and were initiated on durable VAD support at a median of 8 months after completion of cancer treatment (IQR 3.3–43.5 months). Four patients had significant right ventricular dysfunction needing oral pulmonary vasodilator therapy, one patient had a major bleeding complication, and two patients had thromboembolic strokes while on VAD support. Median duration of VAD support was 7.5 months (IQR 3–11.3 months). Two patients underwent VAD explant due to recovery of LV function, one died due to cancer progression, and three underwent heart transplantation.Conclusions: Durable VAD support should be considered as a therapeutic option for children who have advanced HF due to chemotherapy‐induced cardiomyopathy, even within 2 years of completing cancer treatment. A multi‐disciplinary approach is essential for appropriate patient selection prior to implant and to ensure comprehensive care throughout the duration of VAD support.

Data are limited regarding body mass index (BMI) in pediatric patients supported by ventricular assist devices (VAD) and associated clinical outcomes and complications. We performed a retrospective single-center cohort study including patients aged ≤21 years on durable continuous-flow VAD support for ≥30 days from 2009 to 2020. Patients were classified based on BMI percentile at implant using the US Centers for Disease Control and Prevention criteria: underweight (<5th percentile), healthy weight (5th–<85th percentile, reference group), overweight (85th–<95th percentile), and obese (≥95th percentile). Primary outcomes were hospital mortality and length of stay (LOS) after implant. Secondary outcomes included infectious complications and pump thrombosis. Seventy-two patients (58 HeartWare, 13 HeartMateII, 1 HeartMate3) were included. At implant, the study cohort comprised 13% underweight, 53% healthy weight, 18% overweight, and 17% obese. BMI increased across all categories during support, with 29% gaining BMI categories. No patients with obesity reduced their BMI category. At explant, the study cohort comprised 1% underweight, 54% healthy weight, 22% overweight, and 22% obese. There was no significant difference in hospital mortality, postoperative LOS, or pump thrombosis. Patients who were overweight had more frequent non-VAD infections. Patients with obesity required longer duration on VAD support and were less likely to be transplanted. We concluded that pediatric patients on VAD support who are overweight or have obesity do not improve their BMI and instead have significant increase. Larger studies are needed to assess the impact of abnormal BMI on VAD complications in pediatric patients.

Background: Valvular disease in pediatric and young adult donor hearts may be a relative contraindication to graft use. Outcomes following the use of donor hearts with bicuspid aortic valve (BAV) have not been previously reported in children. We describe 4 cases of pediatric heart transplantation (HTx) utilizing a donor heart with a BAV.Case series: Of the 469 HTx included in this study, 4 utilized a donor heart with a BAV. All recipients were female; median age was 11 years (range 0.3 to 19 years). In all cases, the BAV was not discovered until after HTx. All donors were less than 30 years old. The patients were followed for a median of 6 years (range 2 to 9 years) with all patients alive at last follow‐up. Two patients have transitioned to adult care, and 2 patients continue to follow in our clinic. In follow‐up, no patient has required an aortic valve intervention or had infective endocarditis. At last review, no patient had greater than mild aortic insufficiency or more than mild aortic stenosis. Three patients developed mild‐to‐moderate left ventricular hypertrophy in the first year post‐transplant that improved over time. One patient experienced a peri‐operative embolic stroke at time of transplant unrelated to the BAV.Conclusion: On short‐ and intermediate‐term follow‐up, pediatric and young adult donor hearts with BAV demonstrated acceptable graft longevity and valvular function. A functionally normal BAV in a pediatric heart transplant donor should not be a contraindication to organ acceptance.