Advantages, disadvantages and alternatives to using adult heart failure clinical trials to guide pediatric heart failure therapy

2016 ◽  
Vol 43 ◽  
pp. 7-9 ◽  
Author(s):  
Daphne T. Hsu ◽  
Robert E. Shaddy
2019 ◽  
Vol 8 (2) ◽  
pp. 127-127
Author(s):  
Sabine Recla ◽  
Dorle Schmidt ◽  
Thushiha Logeswaran ◽  
Anoosh Esmaeili ◽  
Dietmar Schranz

2021 ◽  
Vol 9 ◽  
Author(s):  
Karla L. Loss ◽  
Robert E. Shaddy ◽  
Paul F. Kantor

Pediatric heart failure (HF) is an important clinical condition with high morbidity, mortality, and costs. Due to the heterogeneity in clinical presentation and etiologies, the development of therapeutic strategies is more challenging in children than adults. Most guidelines recommending drug therapy for pediatric HF are extrapolated from studies in adults. Unfortunately, even using all available treatment, progression to cardiac transplantation is common. The development of prospective clinical trials in the pediatric population has significant obstacles, including small sample sizes, slow recruitment rates, challenging endpoints, and high costs. However, progress is being made as evidenced by the recent introduction of ivabradine and of sacubitril/valsartan. In the last 5 years, new drugs have also been developed for HF with reduced ejection fraction (HFrEF) in adults. The use of well-designed prospective clinical trials will be fundamental in the evaluation of safety and efficacy of these new drugs on the pediatric population. The aim of this article is to review the clinical presentation and management of acute and chronic pediatric heart failure, focusing on systolic dysfunction in patients with biventricular circulation and a systemic left ventricle. We discuss the drugs recently approved for children and those emerging, or in use for adults with HFrEF.


2008 ◽  
Vol 10 (2) ◽  
pp. 125-134 ◽  
Author(s):  
Susan R Foerster ◽  
Charles E Canter

Children ◽  
2021 ◽  
Vol 8 (5) ◽  
pp. 322
Author(s):  
Bibhuti B. Das ◽  
William B. Moskowitz ◽  
Javed Butler

This review discusses the potential drug and device therapies for pediatric heart failure (HF) due to reduced systolic function. It is important to realize that most drugs that are used in pediatric HF are extrapolated from adult cardiology practices or consensus guidelines based on expert opinion rather than on evidence from controlled clinical trials. It is difficult to conclude whether the drugs that are well established in adult HF trials are also beneficial for children because of tremendous heterogeneity in the mechanism of HF in children and variations in the pharmacokinetics and pharmacodynamics of drugs from birth to adolescence. The lessons learned from adult trials can guide pediatric cardiologists to design clinical trials of the newer drugs that are in the pipeline to study their efficacy and safety in children with HF. This paper’s focus is that the reader should specifically think through the pathophysiological mechanism of HF and the mode of action of drugs for the selection of appropriate pharmacotherapy. We review the drug and device trials in adults with HF to highlight the knowledge gap that exists in the pediatric HF population.


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