Pediatric Cardiomyopathies.

2017

855-873

2017 Sep 15

1524-4571

<p>Pediatric cardiomyopathies are rare diseases with an annual incidence of 1.1 to 1.5 per 100 000. Dilated and hypertrophic cardiomyopathies are the most common; restrictive, noncompaction, and mixed cardiomyopathies occur infrequently; and arrhythmogenic right ventricular cardiomyopathy is rare. Pediatric cardiomyopathies can result from coronary artery abnormalities, tachyarrhythmias, exposure to infection or toxins, or secondary to other underlying disorders. Increasingly, the importance of genetic mutations in the pathogenesis of isolated or syndromic pediatric cardiomyopathies is becoming apparent. Pediatric cardiomyopathies often occur in the absence of comorbidities, such as atherosclerosis, hypertension, renal dysfunction, and diabetes mellitus; as a result, they offer insights into the primary pathogenesis of myocardial dysfunction. Large international registries have characterized the epidemiology, cause, and outcomes of pediatric cardiomyopathies. Although adult and pediatric cardiomyopathies have similar morphological and clinical manifestations, their outcomes differ significantly. Within 2 years of presentation, normalization of function occurs in 20% of children with dilated cardiomyopathy, and 40% die or undergo transplantation. Infants with hypertrophic cardiomyopathy have a 2-year mortality of 30%, whereas death is rare in older children. Sudden death is rare. Molecular evidence indicates that gene expression differs between adult and pediatric cardiomyopathies, suggesting that treatment response may differ as well. Clinical trials to support evidence-based treatments and the development of disease-specific therapies for pediatric cardiomyopathies are in their infancy. This compendium summarizes current knowledge of the genetic and molecular origins, clinical course, and outcomes of the most common phenotypic presentations of pediatric cardiomyopathies and highlights key areas where additional research is required.</p>

<p><strong>CLINICAL TRIAL REGISTRATION: </strong>URL: http://www.clinicaltrials.gov. Unique identifiers: NCT02549664 and NCT01912534.</p>

10.1161/CIRCRESAHA.116.309386

Circ. Res.

28912187

Hospital Charges for Pediatric Heart Failure-Related Hospitalizations from 2000 to 2009.

2016

512-8

2016 Mar

1432-1971

<p>Scarce data exist regarding costs of pediatric heart failure-related hospitalizations (HFRH) or how costs have changed over time. Pediatric HFRH costs, due to advances in management, will have increased significantly over time. A retrospective analysis of Healthcare Cost and Utilization Project Kids' Inpatient Database was performed on all pediatric HFRH. Inflation-adjusted charges are used as a proxy for cost. There were a total of 33,189 HFRH captured from 2000 to 2009. Median charges per HFRH rose from $35,079 in 2000 to $72,087 in 2009 (p &lt; 0.0001). The greatest median charges were incurred in patients on extracorporeal membrane oxygenation ($442,134 vs $53,998) or ventricular assist devices ($462,647 vs $55,151). Comorbidities, including sepsis ($207,511 vs $48,995), renal failure ($180,624 vs $52,812), stroke ($198,260 vs $54,974) and respiratory failure ($146,200 vs $48,797), were associated with greater charges (p &lt; 0.0001). Comorbidities and use of mechanical support increased over time. After adjusting for these factors, later year remained associated with greater median charges per HFRH (p &lt; 0.0001). From 2000 to 2009, there has been an almost twofold increase in pediatric HFRH charges, after adjustment for inflation. Although comorbidities and use of mechanical support account for some of this increase, later year remained independently associated with greater charges. Further study is needed to understand potential factors driving these higher costs over time and to identify more cost-effective therapies in this population.</p>

10.1007/s00246-015-1308-0

Pediatr Cardiol

26645995

Use of sirolimus in pediatric heart transplant patients: A multi-institutional study from the Pediatric Heart Transplant Study Group.

2016

2016 Oct 04

1557-3117

<p><strong>BACKGROUND: </strong>Proliferation signal inhibitors, such as sirolimus, are increasingly used in solid-organ transplantation. However, limited data exist on sirolimus-treated pediatric patients. We aimed to describe sirolimus use in pediatric heart transplant patients and test the hypothesis that sirolimus use is associated with improved outcomes.</p>

<p><strong>METHODS: </strong>A retrospective review and propensity-matched analysis of the Pediatric Heart Transplant Study database was performed on patients undergoing primary heart transplantation from 2004 to 2013 with at least 1 year of follow-up comparing patients treated vs not treated with sirolimus at 1 year after transplant. The primary outcome of interest was patient survival, with secondary outcomes including cardiac allograft vasculopathy, rejection, malignancy, and renal insufficiency.</p>

<p><strong>RESULTS: </strong>Between 2004 and 2013, 2,531 patients underwent transplantation. At least 1 year of follow-up was available for 2,080 patients, of whom 144 (7%) were on sirolimus at 1 year post-transplant. Sirolimus-treated and non-treated patients had similar survival in the overall cohorts and in the propensity-matched analysis. The secondary outcomes measures were also similar, including a composite end point of all outcome measures. There was a trend toward increased time to cardiac allograft vasculopathy (p = 0.09) and decreased time to infection (p = 0.05) among sirolimus-treated patients in the overall cohort (p = 0.19) but not in the propensity-matched cohort (p = 0.17).</p>

<p><strong>CONCLUSIONS: </strong>Sirolimus was used in less than 10% of patients at 1 year post-transplant. Overall outcomes of sirolimus treated and non-treated patients were similar with respect to survival and major transplant adverse events. Further study of sirolimus in pediatric heart transplant patients is needed.</p>

10.1016/j.healun.2016.09.009

J. Heart Lung Transplant.

28029575

Pediatric Cardiac Intensive Care Society 2014 Consensus Statement: Pharmacotherapies in Cardiac Critical Care Chronic Heart Failure.

2016

S20-34

2016 Mar

1529-7535

<p><strong>OBJECTIVE: </strong>Heart failure is a serious complication that can occur in patients with a variety of congenital and acquired disorders including congenital heart disease, cardiomyopathy, and myocarditis. Furthermore, heart failure patients comprise an increasing number of ICU admissions. Thus, it is important for those caring for patients with critical cardiovascular disease to have a thorough understanding of the medications used for the treatment of heart failure. The aim of this review is to provide an overview, rationale, indications, and adverse effects of medications used in the treatment of chronic heart failure.</p>

<p><strong>DATA SOURCES: </strong>PubMed, Medline, Cochrane Database of Systemic Reviews.</p>

<p><strong>STUDY SELECTION: </strong>Studies were selected on their relevance for pediatric heart failure. When limited data on pediatric heart failure were available, studies in adult patients were selected.</p>

<p><strong>DATA EXTRACTION: </strong>Relevant findings from studies were selected by the authors.</p>

<p><strong>DATA SYNTHESIS: </strong>The rationale for the efficacy of most heart failure medications used in pediatric patients is extrapolated from studies in adult heart failure. Commonly used medications for chronic heart failure include β-receptor antagonists (e.g., carvedilol and metoprolol), and medications aimed at blocking the renin-angiotensin-aldosterone system (e.g., angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, aldosterone receptor antagonists). In addition, diuretics are useful for symptoms of fluid overload. For patients with impaired perfusion, inotropic agents are useful acutely, but may be associated with worse outcomes when used chronically. Newer medications that have been recently approved in adults (e.g., serelaxin, ivabradine, and neprilysin inhibitor [angiotensin receptor blocker]) may prove to be important in pediatric heart failure.</p>

<p><strong>CONCLUSIONS: </strong>Heart failure patients are in an important population of critically ill children. The pharmacologic approach to these patients is aimed at treating symptoms of congestion and/or poor perfusion and improving long-term outcomes.</p>

10.1097/PCC.0000000000000624

Pediatr Crit Care Med

26945326

Prevalence, predictors, and outcomes of cardiorenal syndrome in children with dilated cardiomyopathy: a report from the Pediatric Cardiomyopathy Registry.

2015

2177-88

2015 Dec

1432-198X

<p><strong>BACKGROUND: </strong>The association of cardiorenal syndrome (CRS) with mortality in children with dilated cardiomyopathy (DCM) is unknown.</p>

<p><strong>METHODS: </strong>With a modified Schwartz formula, we estimated glomerular filtration rates (eGFR) for children ≥1 year of age with DCM enrolled in the Pediatric Cardiomyopathy Registry at the time of DCM diagnosis and annually thereafter. CRS was defined as an eGFR of &lt;90 mL/min/1.73 m(2). Children with and without CRS were compared on survival and serum creatinine concentrations (SCr). The association between eGFR and echocardiographic measures was assessed with linear mixed-effects regression models.</p>

<p><strong>RESULTS: </strong>Of 285 eligible children with DCM diagnosed at ≥1 year of age, 93 were evaluable. CRS was identified in 57 of these 93 children (61.3%). Mean (standard deviation) eGFR was 62.0 (22.6) mL/min/1.73 m(2) for children with CRS and 108.0 (14.0) for those without (P &lt; 0.001); median SCr concentrations were 0.9 and 0.5 mg/dL, respectively (P &lt; 0.001). The mortality hazard ratio of children with CRS versus those with no CRS was 2.4 (95% confidence interval 0.8-7.4). eGFR was positively correlated with measures of left ventricular function and negatively correlated with age.</p>

<p><strong>CONCLUSIONS: </strong>CRS in children newly diagnosed with DCM may be associated with higher 5-year mortality. Children with DCM, especially those with impaired left ventricular function, should be monitored for renal disease.</p>

10.1007/s00467-015-3165-8

Pediatr. Nephrol.

26210985

Clinical Issues and Controversies in Heart Failure and Transplantation.

2016

63-71

2016 Jan

2150-136X

<p>Heart failure is a common problem among children admitted in the intensive care unit and is associated with significant morbidity and mortality. As such, the 2014 meeting of the Pediatric Cardiac Intensive Care Society included a session on Clinical Controversies in Heart Failure and Transplantation. This review contains the summaries of the podium presentations of this session and will cover some of the challenging aspects of caring for these patients including medical and mechanical support, fluid overload states, high-risk populations including those after heart transplantation, and end-of-life considerations.</p>

10.1177/2150135115606622

World J Pediatr Congenit Heart Surg

26714996