Abstract
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.
Highlights
AND CLASSIFICATION OF HEART FAILUREPediatric heart failure (HF) can be defined as a clinical syndrome resulting from ventricular dysfunction, and volume or pressure overload, alone or in combination [1, 2]
The two most common pathophysiological categories resulting in end stage HF in children are cardiomyopathy and congenital heart disease (CHD), each contributing about half of the cases resulting in cardiac transplant, according to recent International Society for Heart and Lung Transplant data [3]
Cardiomyopathy presents with predictable phenotypes, and dilated cardiomyopathy (DCM) or LV non-compaction cardiomyopathy (LVNC) typically manifest clinically as HF with reduced ejection fraction (HFrEF): this term implies by convention, the presence of symptomatic HF, a dilated left ventricle and an LV ejection fraction (EF) of
Summary
Due to the heterogeneity in clinical presentation and etiologies, the development of therapeutic strategies is more challenging in children than adults. 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. In the last 5 years, new drugs have 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
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