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Published on: May 16, 2020
Pediatric dilated cardiomyopathy: a review of current clinical approaches and pathogenesis
Ian Malinow1, Daniel C Fong1, Matthew Miyamoto1
1Division of Cardiovascular Medicine, Department of Medicine, University of Maryland School of Medicine, Baltimore, MD, United States.
Insights
Pediatric dilated cardiomyopathy (DCM) is a rare heart condition with limited treatment options. Understanding its unique genetic and molecular causes is key to developing effective, child-specific therapies.
Area of Science:
- Cardiology
- Pediatric Medicine
- Genetics
Background:
- Pediatric dilated cardiomyopathy (DCM) is a severe condition affecting children, often leading to heart transplant or death.
- Current diagnostic methods rely on clinical and echocardiographic findings, with prognosis influenced by age, etiology, and cardiac function.
- Limited therapeutic options exist, and many adult treatments lack proven efficacy in children.
Purpose of the Study:
- To review the biological pathogenesis of pediatric DCM, distinguishing it from adult forms.
- To discuss current clinical guidelines and emerging therapeutic strategies for pediatric DCM.
- To highlight the need for child-specific treatment approaches based on a better understanding of disease mechanisms.
Main Methods:
- Review of established biological pathogenesis in pediatric DCM.
- Analysis of current clinical guidelines for diagnosis and management.
- Exploration of promising therapeutic avenues and recent research findings.
Main Results:
- Pediatric DCM has a significant genetic component, with links to sarcomere and cytoskeleton gene variants.
- Infantile DCM may involve distinct etiologies, such as impaired postnatal cardiac maturation.
- Approximately two-thirds of pediatric DCM cases are idiopathic, indicating a need for further research.
Conclusions:
- Improved understanding of pediatric DCM pathogenesis is crucial for developing novel, child-specific treatments.
- Addressing the unique genetic and molecular aspects of pediatric DCM can improve patient prognosis and quality of life.
- Further research is essential to unravel the complexities of this rare cardiovascular condition in children.
Abstract:
Pediatric dilated cardiomyopathy (DCM) is a rare, yet life-threatening cardiovascular condition characterized by systolic dysfunction with biventricular dilatation and reduced myocardial contractility. Therapeutic options are limited with nearly 40% of children undergoing heart transplant or death within 2 years of diagnosis. Pediatric patients are currently diagnosed based on correlating the clinical picture with echocardiographic findings. Patient age, etiology of disease, and parameters of cardiac function significantly impact prognosis. Treatments for pediatric DCM aim to ameliorate symptoms, reduce progression of disease, and prevent life-threatening arrhythmias. Many therapeutic agents with known efficacy in adults lack the same evidence in children. Unlike adult DCM, the pathogenesis of pediatric DCM is not well understood as approximately two thirds of cases are classified as idiopathic disease. Children experience unique gene expression changes and molecular pathway activation in response to DCM. Studies have pointed to a significant genetic component in pediatric DCM, with variants in genes related to sarcomere and cytoskeleton structure implicated. In this regard, pediatric DCM can be considered pediatric manifestations of inherited cardiomyopathy syndromes. Yet exciting recent studies in infantile DCM suggest that this subset has a distinct etiology involving defective postnatal cardiac maturation, such as the failure of programmed centrosome breakdown in cardiomyocytes. Improved knowledge of pathogenesis is central to developing child-specific treatment approaches. This review aims to discuss the established biological pathogenesis of pediatric DCM, current clinical guidelines, and promising therapeutic avenues, highlighting differences from adult disease. The overarching goal is to unravel the complexities surrounding this condition to facilitate the advancement of novel therapeutic interventions and improve prognosis and overall quality of life for pediatric patients affected by DCM.
