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Review
. 2023 Mar 31;12(3):503-513.
doi: 10.21037/tp-22-431. Epub 2023 Feb 16.

Narrative review of pediatric heart failure in the age of precision medicine

Nishma Valikodath  1 , Justin Godown  2 , Aryaz Sheybani  3
Affiliations
Review

Narrative review of pediatric heart failure in the age of precision medicine

Nishma Valikodath et al. Transl Pediatr. .

Abstract

Background and objective: Improvement in the affordability and convenience of genetic testing has rapidly expanded the understanding of the mechanistic causes of various pediatric cardiomyopathies. Concurrently, new therapies are being developed to better-target specific pathologies as opposed to classic therapies that treat the maladaptive processes of chronic heart failure. This review will discuss the advances in genetic testing and specific therapies that have been shown to benefit or potentially benefit genetically distinct subsets of the pediatric population with heart failure or at risk of developing heart failure.

Methods: We undertook a comprehensive database search (January 2000-August 2022) of PubMed, utilizing terms 'pediatric', 'cardiomyopathy', 'heart failure', 'genetics', and 'precision medicine'. Additional notable studies were obtained from ClinicalTrials.gov. Studies published in English that examine genetic basis and treatment modalities of pediatric heart failure.

Key content and findings: New and investigational therapies for hypertrophic cardiomyopathies associated with obstruction or Noonan syndrome, Fabry cardiomyopathy, Barth syndrome, Duchenne muscular dystrophy, single ventricle failure, and heart failure in specific demographics are discussed.

Conclusions: The rapid expansion of the genetic understanding of cardiomyopathy and heart failure as well as tailored therapies to specific molecular causes holds great promise for the future of pediatric heart failure treatment. Whereas conventional heart failure therapies target the maladaptive remodeling response that leads to worsening of heart failure, these therapies target the molecular causes of cardiomyopathy and heart failure in certain populations allowing for a potential to more significantly impact the clinical trajectory of pediatric heart failure.

Keywords: Heart failure; pediatric; precision medicine.

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Conflict of interest statement

Conflicts of Interest: All authors have completed the ICMJE uniform disclosure form (available at https://tp.amegroups.com/article/view/10.21037/tp-22-431/coif). JG serves as an unpaid editorial board member of Translational Pediatrics from January 2023 to December 2024. He also serves as a consultant to Daiichi Sankyo. The other authors have no conflicts of interest to declare.

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