Treating Children With Idiopathic Dilated Cardiomyopathy (from the Pediatric Cardiomyopathy Registry)

Abstract

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]...

Idiopathic Cardiomyopathies in Korean Children - 9-Year Korean Multicenter Study -

“…A study of the epidemiologic characteristics of idiopathic CMP from the pediatric CMP registry in the USA revealed that approximately 5% of patients underwent cardiac transplantation, 2% IABP, 2% ECMO, and 1% VAD; however, the medical outcomes in these children did not show improvement in the previous several decades. 19 There have been reports on various therapeutic trials in addition to medical therapy of CMP prior to cardiac transplantation. 20- 22 Although we could not collect all of the data on CMP patients nationwide, the numbers of patients treated with surgical mechanical support have shown an increase in recent years and these therapeutic trials and experience might have an impact on the prognosis of primary CMP in the future.…”

Current management of pediatric dilated cardiomyopathy

“…Many pediatric centersroutinely supplement their PDCM patients with carnitine [10,17], regardless of cause. This practice is contrary to American and European adult heart failure guidelines, in which routine use of nutritional, hormonal supplements, or both for the treatment of heart failure is not recommended [8,9]. The challenge for pediatric cardiologists treating PDCM is to ensure patients who truly will benefit from such therapy will be identified.…”

“…A number of these studies [46][47][48][49][50][51] have demonstrated short-term efficacy of ACE inhibitors for systemic ventricular dysfunction in children. It would appear that only approximately 65-70% of patients with an idiopathic dilated cardiomyopathy receive ACE inhibitors, and that proportion has remained stable from 1990 to 2006 [9]. ARBs have been studied in children with hypertension [52] but not heart failure.…”

“…This study has led to the development of clinical guidelines for the treatment of left ventricular dysfunction in adults, which have been recently revised by both the American Heart Association [7] and the European Society of Cardiology [8]. However, a recent study from the Pediatric Cardiomyopathy Registry (PCMR) [9] suggests that treatment of PDCM has not changed substantially over the past 25 years. The purpose of this review is to discuss recent developments in the treatment of PDCM and how adult guidelines can be reconciled with PDCM.…”

Clinical management of dilated cardiomyopathy: current knowledge and future perspectives

“…[15] Left ventricular reverse remodeling (LVRR) appears to predict a lower rate of major cardiac adverse events independently from other factors, such as NYHA class or FMR. [12] It is mostly induced within the first 6 months of optimal medical treatment, [16] but can be completed at 24 months.…”