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Preventing pediatric cardiomyopathy: a 2015 outlook
Paul F Kantor1, Jake A Kleinman2, Thomas D Ryan3
1a Pediatric Cardiology , Stollery Children's Hospital , Edmonton , Alberta , Canada.
Insights
Preventing pediatric cardiomyopathies requires addressing root causes from molecular to population levels. Advances in gene therapy and diagnostics are crucial for future prevention strategies.
Area of Science:
- Pediatric Cardiology
- Genetics
- Public Health
Background:
- Cardiomyopathies in children represent a significant and increasing global health burden.
- These conditions can be primary cardiac disorders or secondary to systemic diseases, with limited disease-altering treatments available.
- A focused review on prevention is therefore essential.
Purpose of the Study:
- To review the prevention of cardiomyopathy in children.
- To address root causes at molecular, clinical, and population levels.
- To highlight areas for future research and improved diagnostics.
Main Methods:
- Review of recent basic research findings.
- Analysis of advancements in gene-targeted therapy, antiviral therapies, and cardiotoxic drug modification.
- Discussion of public health initiatives, vaccine development, and treatment adoption.
Main Results:
- Promising basic research returns in gene therapy, antiviral treatments, and managing cardiotoxic drug effects.
- Significant work is still needed in vaccine development, public health, and treatment accessibility.
- Refinement and broader availability of diagnostic methods, including imaging and genetic testing, are critical.
Conclusions:
- Effective prevention of childhood cardiomyopathies necessitates a multi-level approach.
- Continued research, improved diagnostics, and public health strategies are vital.
- Leveraging registries for patient phenotyping and longitudinal follow-up is key to advancing knowledge.
Abstract:
Cardiomyopathies in children encompass a broad range of diseases, both genetic and acquired, which manifest as a primary cardiac disorder or as a cardiomyopathy secondary to systemic disease. The burden of this group of disorders is substantial, and growing on a global scale. The availability of disease altering treatments is limited, and therefore a focused review on the prevention of cardiomyopathies is justified. In this review, we address the prevention of cardiomyopathy in children by dealing with the root causes of disease at a molecular, clinical and population level. Recent years have yielded promising returns in basic research related to gene-targeted therapy, specific anti-viral therapies and modification of the effects of cardiotoxic drugs. Much work remains to be done in the fields of vaccine development, public health and adoption of available treatments. Effective research in this field will require that diagnostic methods are both refined, and made available more broadly, from imaging to gene testing. Much of our knowledge today is derived from the use of registries, which have successfully catalogued the detailed phenotype of affected patients, and provided long-term longitudinal follow up of affected individuals.
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