Chronic heart failure (HF) in children and adolescents with congenital heart disease (CHD) presents an increasingly prevalent and formidable challenge within the field of pediatric cardiology.^1-3 Characterized by intricate pathophysiological mechanisms and an extensive array of associated comorbidities, pediatric HF necessitates a comprehensive, standardized approach to both diagnosis and management. Although significant strides have been made in the care of adult patients with HF, the domain of pediatric HF in the context of CHD remains insufficiently explored, resulting in critical gaps in both understanding and therapeutic strategies.

This scientific statement from the American Heart Association (AHA) provides an in-depth examination of the unique epidemiological patterns, pathophysiological processes, clinical staging, and management complexities inherent to HF in this vulnerable population.⁴ It underscores the urgent need for the development of innovative therapeutic modalities and the establishment of tailored care protocols that address the specific and evolving needs of pediatric patients with CHD. The following are key points to remember about the AHA scientific statement:

1. Epidemiology and Burden: The incidence and prevalence of HF is rising among children and adolescents with CHD. CHD is the leading cause for pediatric HF-related emergency department visits and hospitalizations.^1-3
2. Pathophysiology: HF arises from unique factors in CHD, including chronic pressure/volume overload, ischemic damage, and postoperative complications like low cardiac output. In specific lesions, such as single-ventricle physiology, mitochondrial dysfunction and oxidative stress further contribute to disease progression.
3. Definitions and Staging: Standardizing HF definition and staging in patients with CHD is critical. Clear staging frameworks, incorporating ventricular function, biomarkers, and clinical symptoms, are essential for treatment and research standardization. Like adults, children and adolescents with CHD experience systolic (HF with reduced ejection fraction) and diastolic HF (HF with preserved ejection fraction). Assessment of systolic function in those with systemic right ventricle or indeterminate ventricular morphology is challenging. For those with CHD, using provocation (by exercise or fluid challenge) may help unmask occult diastolic dysfunction (Figure 1).

   Figure 1: Stages of Heart Failure and Potential Therapies in Patients With Congenital Heart Disease

   

   Reprinted with permission from Amdani S, Conway J, George K, et al. Evaluation and management of chronic heart failure in children and adolescents with congenital heart disease: a scientific statement from the American Heart Association. Circulation. 2024;150(2):e33-e50. doi:10.1161/CIR.0000000000001245. © 2024 American Heart Association, Inc.
   ACE = angiotensin-converting enzyme; ARB = angiotensin receptor blocker; ARNI = angiotensin receptor–neprilysin inhibition; HD = heart disease; LV = left ventricular; NYHA = New York Heart Association; RV = right ventricular; and VAD = ventricular assist device.
   *Caution advised in presence of hepatic or renal dysfunction.

4. Comorbidities and Impact: Arrhythmias, cyanosis, and pulmonary hypertension exacerbate HF in CHD. End-organ dysfunction, including kidney and hepatic injury, also worsens outcomes, highlighting the need for comprehensive, multidisciplinary care.
5. Nutritional Support: Malnutrition and growth failure are common in pediatric HF due to increased metabolic demands and poor intake. Nutritional interventions, including high-caloric diets and supplementation, have the potential to improve strength and resilience against HF progression.
6. Pharmacological Management: Evidence supporting HF therapies in CHD is limited.^5-7 Although renin-angiotensin system inhibitors, beta-blockers, mineralocorticoid receptor antagonists, and diuretics are frequently used, their benefits are not established. Novel agents, such as sodium-glucose cotransporter-2 inhibitors and ivabradine, show promise but require further validation in this population.
7. Advanced Therapies: Mechanical circulatory support, including ventricular assist devices, and heart transplantation are critical for patients with end-stage HF (stage D).^8,9 The timing of these interventions is crucial to optimize outcomes, especially in those with single-ventricle anatomy.
8. Surveillance and Follow-Up: Continuous evaluation of HF severity is vital. A framework incorporating growth parameters, exercise tolerance, biomarker levels, hemodynamic parameters, functional limitation, and hospitalization frequency can allow clinicians to evaluate HF severity and adjust management accordingly.
9. Knowledge Gaps: Significant research gaps remain in understanding the interplay between CHD anatomy, HF progression, and therapy response.¹⁰ This concise AHA statement captures actionable insights for managing chronic HF in pediatric patients with CHD, emphasizing the importance of integrated care approaches tailored to their unique pathophysiology.
10. Future Directions: Collaborative, multi-institutional studies using real-world datasets and advanced analytics are critical to developing robust, evidence-based care protocols for this high-risk population.

References

1. Amdani S, Marino BS, Rossano J, Lopez R, Schold JD, Tang WHW. Burden of pediatric heart failure in the United States. J Am Coll Cardiol. 2022;79(19):1917-1928. doi:10.1016/j.jacc.2022.03.336
2. Burstein DS, Shamszad P, Dai D, et al. Significant mortality, morbidity and resource utilization associated with advanced heart failure in congenital heart disease in children and young adults. Am Heart J. 2019;209:9-19. doi:10.1016/j.ahj.2018.11.010
3. Amdani S, Lopez R, Schold JD, Tang WHW. 30- and 60-day readmission rates for children with heart failure in the United States. JACC Heart Fail. 2024;12(1):83-96. doi:10.1016/j.jchf.2023.08.029
4. Amdani S, Conway J, George K, et al. Evaluation and management of chronic heart failure in children and adolescents with congenital heart disease: a scientific statement from the American Heart Association. Circulation. 2024;150(2):e33-e50. doi:10.1161/CIR.0000000000001245
5. Shaddy RE, Boucek MM, Hsu DT, et al. Carvedilol for children and adolescents with heart failure: a randomized controlled trial. JAMA. 2007;298(10):1171-1179. doi:10.1001/jama.298.10.1171
6. Hsu DT, Zak V, Mahony L, et al. Enalapril in infants with single ventricle: results of a multicenter randomized trial. Circulation. 2010;122(4):333-340. doi:10.1161/CIRCULATIONAHA.109.927988
7. Shaddy R, Burch M, Kantor PF, et al. Sacubitril/valsartan in pediatric heart failure (PANORAMA-HF): a randomized, multicenter, double-blind trial. Circulation. 2024;150(22):1756-1766. doi:10.1161/CIRCULATIONAHA.123.066605
8. Lorts A, Conway J, Schweiger M, et al. ISHLT consensus statement for the selection and management of pediatric and congenital heart disease patients on ventricular assist devices endorsed by the American Heart Association. J Heart Lung Transplant. 2021;40(8):709-732. doi:10.1016/j.healun.2021.04.015
9. Townsend M, Karamlou T, Boyle G, et al. Waitlist outcomes for children with congenital heart disease: lessons learned from over 5000 heart transplant listings in the United States. J Card Fail. 2022;28(6):982-990. doi:10.1016/j.cardfail.2022.03.004
10. Amdani S, Auerbach SR, Bansal N, et al. Research gaps in pediatric heart failure: defining the gaps and then closing them over the next decade. J Card Fail. 2024;30(1):64-77. doi:10.1016/j.cardfail.2023.08.026