Pediatric cardiomyopathies, which are rare but serious disorders of the muscles of the heart, affect at least one in every 100,000 children in the USA. Approximately 40% of children with symptomatic cardiomyopathy undergo heart transplantation or die from cardiac complications within 2 years. However, a significant number of children suffering from cardiomyopathy are surviving into adulthood, making it an important chronic illness for both pediatric and adult clinicians to understand. The natural history, risk factors, prevalence and incidence of this pediatric condition were not fully understood before the 1990s. Questions regarding optimal diagnostic, prognostic and treatment methods remain. Children require long-term follow-up into adulthood in order to identify the factors associated with best clinical practice including diagnostic approaches, as well as optimal treatment approaches. In this article, we comprehensively review current research on various presentations of this disease, along with current knowledge about their causes, treatments and clinical outcomes.

In 40% of children with symptomatic idiopathic dilated cardiomyopathy (IDC), medical therapy fails within 2 years of diagnosis. Strong evidence-based therapies are not available for these children, and how evidence-based therapies for adults with IDC should be applied to children is unclear. Using data from the National Heart, Lung, and Blood Institute's Pediatric Cardiomyopathy Registry, we compared practice patterns of initial therapies for children with IDC diagnosed between 1990 and 1995 (n=350) and between 2000 and 2006 (n=219). At diagnosis, 73% had symptomatic heart failure, and 7% had one or more family members with IDC. Anti-heart failure medications were most commonly prescribed initially. Anti-heart failure medication use was similar across both time periods (84% and 87%, respectively), as was angiotensin-converting enzyme inhibitor (ACEI) use (66% and 70%, respectively). These medications were used more commonly in children with greater left ventricular dilation and poorer left ventricular fractional shortening and functional class (P<0.001). Beta-blocker use ranged from 4% to 18% over the two time periods. Treatments for pediatric IDC have changed little over the past 25 years. Anti-heart failure medications remain the most common treatment, and they are often given to children with asymptomatic left ventricular dysfunction. Children with asymptomatic left ventricular dysfunction are often not offered ACEIs without echocardiographic evidence of advanced disease. Therapeutic clinical trials are strongly indicated because practice variation is substantial and medical outcomes in these children have not improved in the past several decades. KeywordsPediatrics; Cardiomyopathy; Heart Failure; Anti-heart failure Therapy © 2009 Excerpta Medica, Inc. All rights reserved.Corresponding Author: Steven E. Lipshultz, MD, Department of Pediatrics, University of Miami Miller School of Medicine, Medical Campus-MCCD-D820, 1601 NW 12th Avenue, Miami, FL 33136, slipshultz@med.miami.edu. Publisher's Disclaimer: This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final citable form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain. In the absence of evidence-based standards, the clinical treatment of children with idiopathic dilated cardiomyopathy (IDC) and heart failure (HF) varies widely. We examined the medical therapies offered at presentation to children with IDC who were enrolled in the retrospective arm of the PCMR database to identify and characterize treatment patterns in managing childhood HF. To examine management trends over time, we compared the rates of pharmacological therapy to a later cohort from the prospective arm of the registry. [1][2][3][4][5]...

Background-Increasing serum levels of N-terminal pro-hormone brain natriuretic peptide (NTproBNP) are associated with worsening heart failure (HF) in adults. We determined whether changes in NT-proBNP level are associated with changes in symptoms and left ventricular (LV) systolic function and remodeling in children with HF secondary to dilated cardiomyopathy.