ArticleAnnals of pediatric cardiology
Advances and unmet needs in pharmacologic therapy for pediatric heart failure: Insights from the 2025 International Society for Heart and Lung Transplantation Guidelines.
Article in Annals of pediatric cardiology. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Backgrounds: Pediatric heart failure (PHF), most commonly resulting from cardiomyopathies, congenital heart disease, or acquired cardiac disorders, remains a major cause of childhood morbidity and mortality. The 2025 International Society for Heart and Lung Transplantation (ISHLT) guidelines provide the most comprehensive update in over a decade, offering a contemporary framework for pharmacologic management when underscoring persistent gaps in pediatric-specific evidence. Methods: This narrative review synthesizes the 2025 ISHLT Guidelines alongside contemporary pediatric and adult HF guidance, emphasizing pharmacologic recommendations, areas of extrapolation from adult trials, and persistent evidence gaps across diverse PHF phenotypes. Results: The updated guideline introduces an ejection fraction (EF)-based classification system and refines pharmacologic recommendations for acute and chronic PHF. It reaffirms Class I support for foundational neurohormonal therapies in PHF with reduced EF, incorporates randomized control trial-based recommendations for sacubitril-valsartan and ivabradine, and offers limited, extrapolated recommendations for agents such as sodium-glucose cotransporter-2 inhibitors, mineralocorticosteroid receptor antagonists, and soluble guanylate cyclase stimulators. The guideline also outlines phenotype-specific considerations for high-risk groups, including congenital heart disease and cancer therapy-related cardiomyopathy. Persistent unmet needs include the absence of disease-modifying therapies for PHF with preserved EF and insufficient evidence to guide management of complex congenital and syndromic conditions. Conclusion: The 2025 ISHLT Guidelines provide a structured, contemporary approach to pharmacologic therapy in PHF when clearly delineating areas where evidence remains limited. Addressing these limitations will require coordinated multicenter research, real-world data integration, and learning health systems capable of translating emerging therapies into evidence-based pediatric practice.
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