Current management of pediatric dilated cardiomyopathy

Silva, Jennifer N. Avari; Canter, Charles E.

doi:10.1097/hco.0b013e328335b220

Cited by 14 publications

(8 citation statements)

References 96 publications

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“…As a refractory life-threatening pediatric condition which eventually leads to heart failure, heart transplantation remains the ultimate treatment for DCM [ 5 ]. Due to the invasive and costly nature of heart transplantation, alternative therapies with higher accessibility are imperative for pediatric DCM.…”

Section: Introductionmentioning

confidence: 99%

Intramuscular injection of human umbilical cord-derived mesenchymal stem cells improves cardiac function in dilated cardiomyopathy rats

Mao

Wang

et al. 2017

Stem Cell Res Ther

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BackgroundStem cells provide a promising candidate for the treatment of the fatal pediatric dilated cardiomyopathy (DCM). This study aimed to investigate the effects of intramuscular injection of human umbilical cord-derived mesenchymal stem cells (hUCMSCs) on the cardiac function of a DCM rat model.MethodsA DCM model was established by intraperitoneal injections of doxorubicin in Sprague-Dawley rats. hUCMSCs at different concentrations or cultured medium were injected via limb skeletal muscles, with blank medium injected as the control. The rats were monitored for 4 weeks, meanwhile BNP, cTNI, VEGF, HGF, GM-CSF, and LIF in the peripheral blood were examined by ELISA, and cardiac function was monitored by echocardiography (Echo-CG). Finally, the expression of IGF-1, HGF, and VEGF in the myocardium was examined by histoimmunochemistry and real-time PCR, and the ultrastructure of the myocardium was examined by electron microscopy.ResultsInjection of hUCMSCs markedly improved cardiac function in the DCM rats by significantly elevating left ventricular ejection fraction (LVEF) and left ventricular fraction shortening (LVFS). The BNP and cTNI levels in the peripheral blood were reduced by hUCMSCs, while HGF, LIF, GM-CSF, and VEGF were increased by hUCMSCs. Expression of IGF-1, HGF, and VEGF in the myocardium from the DCM rats was significantly increased by hUCMSC injection. Furthermore, hUCMSCs protected the ultrastructure of cardiomyocytes by attenuating mitochondrial swelling and maintaining sarcolemma integrity.ConclusionsIntramuscular injection of UCMSCs can improve DCM-induced cardiac function impairment and protect the myocardium. These effects may be mediated by regulation of relevant cytokines in serum and the myocardium.Electronic supplementary materialThe online version of this article (doi:10.1186/s13287-017-0472-y) contains supplementary material, which is available to authorized users.

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Section: Introductionmentioning

confidence: 99%

Intramuscular injection of human umbilical cord-derived mesenchymal stem cells improves cardiac function in dilated cardiomyopathy rats

Mao

Wang

et al. 2017

Stem Cell Res Ther

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“…The treatment strategies for children with asymptomatic heart failure and cardiomyopathy are, in general, similar to those recommended for adults with heart failure [26, 27]. They include ACE inhibitors and β blockers, in addition to diuretics and even Digoxin.…”

Section: Discussionmentioning

confidence: 99%

From discrete dilated cardiomyopathy to successful cardiac transplantation in congenital disorders of glycosylation due to dolichol kinase deficiency (DK1-CDG)

et al. 2012

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Congenital disorders of glycosylation are a growing group of inborn errors of protein glycosylation. Cardiac involvement is frequently observed in the most common form, PMM2-CDG, especially hypertrophic cardiomyopathy. Dilated cardiomyopathy, however, has been only observed in a few CDG subtypes, usually with a lethal outcome. We report on cardiac pathology in nine patients from three unrelated Israeli families, diagnosed with dolichol kinase deficiency, due to novel, homozygous DK1 gene mutations. The cardiac symptoms varied from discrete, mild dilation to overt heart failure with death. Two children died unexpectedly with acute symptoms of heart failure before the diagnosis of DK1-CDG and heart transplantation could take place. Three other affected children with mild dilated cardiomyopathy at the time of the diagnosis deteriorated rapidly, two of them within days after an acute infection. They all went through successful heart transplantation; one died unexpectedly and 2 others are currently (after 1–5 years) clinically stable. The other 4 children diagnosed with mild dilated cardiomyopathy are doing well on supportive heart failure therapy. In most cases, the cardiac findings dominated the clinical picture, without central nervous system or multisystem involvement, which is unique in CDG syndrome. We suggest to test for DK1-CDG in patients with dilated cardiomyopathy. Patients with discrete cardiomyopathy may remain stable on supportive treatment while others deteriorate rapidly. Our paper is the first comprehensive study on the phenotype of DK1-CDG and the first successful organ transplantation in CDG syndrome.

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“…There is fairly good evidence for the efficacy of milrinone, levosimendan, or nesiritide used individually in the pediatric population (5–10). Studies conducted to date evaluate the use of each individual drug, sometimes in combination with catecholamines, and such studies were performed mainly in adults.…”

Section: Discussionmentioning

confidence: 99%

“…A paradigm shift has occurred in the treatment of chronic heart failure in the adult population (4), focusing on cardioprotection using β‐blockers, preload and after load reduction, and the inhibition of apoptosis. This new strategy has not found great acceptance in the treatment of pediatric patients (5,6). At our center, all patients with dilated cardiomyopathy receive angiotensin‐converting enzyme inhibitors, beta‐blockers, and diuretics.…”

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confidence: 99%

How Mechanical Circulatory Support Helps Not to Need It-New Strategies in Pediatric Heart Failure

et al. 2011

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During the past 3 years, seven potential candidates for mechanical circulatory support (MCS) were treated at our center. Ultimately, only one of them needed MCS (extracorporeal membrane oxygenation [ECMO] for 16 days), although 5 years earlier, all would have been considered for MCS at our center. Seven consecutive patients were seen in this period: four toddlers (three suffering from fulminant myocarditis and one with dilated cardiomyopathy associated with spongy myocardium) and three adolescents (two with postmyocarditis cardiomyopathy and one with hypertrophic cardiomyopathy and severe restrictive dysfunction after an ischemic event with cardiopulmonary resuscitation [stunned heart]). All patients presented in acute cardiocirculatory decompensation. All were admitted to the intensive care unit (ICU); all but one were sedated and intubated. A combination of levosimendan, milrinone, and nesiritide was administered to all patients. Use of catecholamines was kept short (<48 h in six individuals). MCS (ECMO, Berlin Heart Excor Pediatric, and Heartware) was always available. MCS initiation was indicated in only one patient, who was developing progressive multiorgan failure (MOF). The three toddlers with myocarditis recovered with complete normalization of myocardial function within 6 months. The fourth toddler is still at the ICU while waiting for transplantation. The three adolescents were listed with high urgency for heart transplantation, and all received a graft within 3 weeks. The adolescent with the stunned heart developed progressive MOF and was successfully supported with ECMO until transplantation. All six patients with completed course were discharged home in New York Heart Association Heart Failure Functional Classification System I condition without neurological deficits. Combined use of levosimendan, milrinone, and nesiritide, avoidance of catecholamines as much as possible, and MCS as backup are the new strategies at our center. This cardioprotective approach gives excellent outcome at lower risk and better cost-effectiveness in our pediatric patients with acute heart failure. Pediatric trials are recommended to evaluate combined use of newer cardioprotective drugs.

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Current management of pediatric dilated cardiomyopathy

Abstract: The robust development of new therapies for adult heart failure has been successfully applied to children with dilated cardiomyopathy. Therapies for severe, intractable heart failure have been more widely utilized than therapies for mild-to-moderate heart failure.

Cited by 14 publications

References 96 publications

Intramuscular injection of human umbilical cord-derived mesenchymal stem cells improves cardiac function in dilated cardiomyopathy rats

Intramuscular injection of human umbilical cord-derived mesenchymal stem cells improves cardiac function in dilated cardiomyopathy rats

From discrete dilated cardiomyopathy to successful cardiac transplantation in congenital disorders of glycosylation due to dolichol kinase deficiency (DK1-CDG)

How Mechanical Circulatory Support Helps Not to Need It-New Strategies in Pediatric Heart Failure

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