Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2025 May 29:9:100292.
doi: 10.1016/j.jhlto.2025.100292. eCollection 2025 Aug.

The state of the art in medical therapies for pediatric heart failure

Humera Ahmed  1 , Joseph W Rossano  1
Affiliations
Review

The state of the art in medical therapies for pediatric heart failure

Humera Ahmed et al. JHLT Open. .

Abstract

Pediatric heart failure is a rare but serious condition affecting children with congenital heart disease and various forms of cardiomyopathy. The treatment paradigm for pediatric heart failure has historically been shaped by expert consensus guidelines, largely informed by the results of adult heart failure trials. Recently, however, there has been an increased focus on pediatric-specific drug development and clinical trials. Medications such as digoxin, beta-blockers, and renin-angiotensin-aldosterone system inhibitors have been explored in children, but responses can vary based on the underlying heart disease. Newer treatments such as sacubitril-valsartan and sodium-glucose cotransporter 2 inhibitors show promise, but more data are needed to determine their safety and efficacy in young children. This article explores the current state of medical therapy for chronic pediatric heart failure, highlighting the evolution of treatment strategies and the novel therapies under exploration.

Keywords: cardiomyopathy; clinical trials; congenital heart disease; gene therapy; heart failure; pediatric cardiology; pharmacotherapy.

PubMed Disclaimer

Conflict of interest statement

The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Joseph Rossano reports a relationship with Merck and Co. Inc. that includes consulting or advisory. Joseph Rossano reports a relationship with Bayer Corporation that includes consulting or advisory. Joseph Rossano reports a relationship with Bristol Myers Squibb Co. that includes consulting or advisory. Joseph Rossano reports a relationship with CRI Biotech that includes consulting or advisory. Joseph Rossano reports a relationship with Asklepios BioPharmaceutical Inc. that includes consulting or advisory. The other authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Figures

Figure 1
Figure 1
The mechanism of heart failure modulation with the use of SGLT2i. The exact mechanism of heart failure modulation with the use of sodium-glucose cotransporter 2 inhibitors is not well known, but is postulated to be multifactorial. Glucosuria results in decreased weight, decreased serum glucose with a potential resultant decrease in inflammation, and increased lipolysis, all of which improve cardiometabolic health. Glucosuria stimulates osmotic diuresis, leading to augmented clearance of intercellular free water. NHE1 inhibition may reduce intracellular sodium load and restore cardiac mitochondrial function. Decreased intraglomerular pressure and decreased hyperfiltration may decrease the effects of cardiorenal syndrome. CKD, chronic kidney disease; NHE1, sarcolemmal Na+/H+ exchanger isoform 1; NHE3, tubular Na+/H+ exchanger isoform 3; DKD, diabetic kidney disease.
See this image and copyright information in PMC

Similar articles

See all similar articles

References

    1. Kantor P.F., Lougheed J., Dancea A., et al. Presentation, diagnosis, and medical management of heart failure in children: Canadian Cardiovascular Society guidelines. Can J Cardiol. 2013;29:1535–1552. doi: 10.1016/j.cjca.2013.08.008. - DOI - PubMed
    1. Ahmed H., VanderPluym C. Medical management of pediatric heart failure. Cardiovasc Diagn Ther. 2021;11:323–335. - PMC - PubMed
    1. Burstein D.S., 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. - DOI - PubMed
    1. Rossano J.W., Kim J.J., Decker J.A., et al. Prevalence, morbidity, and mortality of heart failure-related hospitalizations in children in the United States: a population-based study. J Card Fail. 2012;18:459–470. doi: 10.1016/j.cardfail.2012.03.001. - DOI - PubMed
    1. Lipshultz S.E., Sleeper L.A., Towbin J.A., et al. The incidence of pediatric cardiomyopathy in two regions of the United States. N Engl J Med. 2003;348:1647–1655. doi: 10.1056/NEJMoa021715. - DOI - PubMed

LinkOut - more resources