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Pediatric cardiomyopathies: causes, epidemiology, clinical course, preventive strategies and therapies
Steven E Lipshultz1, Thomas R Cochran, David A Briston
1Department of Pediatrics, University of Miami Miller School of Medicine, 1601 NW 12th Avenue, 9th Floor, Miami, FL 33136, USA.
Insights
Pediatric cardiomyopathies are rare heart muscle disorders in children. Many survivors live into adulthood, requiring ongoing research for optimal diagnosis and treatment.
Area of Science:
- Cardiology
- Pediatric Medicine
- Genetics
Background:
- Pediatric cardiomyopathies are rare but serious heart muscle disorders affecting at least 1 in 100,000 children in the USA.
- Approximately 40% of symptomatic children with cardiomyopathy face heart transplantation or cardiac death within two years.
- Increasing survival rates into adulthood highlight the need for understanding pediatric cardiomyopathy as a chronic illness.
Purpose of the Study:
- To comprehensively review current research on pediatric cardiomyopathies.
- To elucidate the causes, various presentations, treatments, and clinical outcomes of these conditions.
- To address remaining questions regarding optimal diagnostic, prognostic, and treatment strategies.
Main Methods:
- Comprehensive literature review of pediatric cardiomyopathy research.
- Analysis of studies focusing on natural history, risk factors, prevalence, and incidence.
- Synthesis of data on diagnostic approaches, treatment modalities, and long-term follow-up.
Main Results:
- Significant advancements in understanding pediatric cardiomyopathies have occurred since the 1990s.
- Knowledge gaps persist regarding optimal diagnostic, prognostic, and treatment methods.
- Long-term follow-up is crucial for identifying factors associated with best clinical practice.
Conclusions:
- Pediatric cardiomyopathies represent a critical chronic illness requiring integrated pediatric and adult care.
- Further research is essential to refine diagnostic criteria and therapeutic interventions.
- Understanding the long-term trajectory and influencing factors is key to improving patient outcomes.
Abstract:
Pediatric cardiomyopathies, which are rare but serious disorders of the muscles of the heart, affect at least one in every 100,000 children in the USA. Approximately 40% of children with symptomatic cardiomyopathy undergo heart transplantation or die from cardiac complications within 2 years. However, a significant number of children suffering from cardiomyopathy are surviving into adulthood, making it an important chronic illness for both pediatric and adult clinicians to understand. The natural history, risk factors, prevalence and incidence of this pediatric condition were not fully understood before the 1990s. Questions regarding optimal diagnostic, prognostic and treatment methods remain. Children require long-term follow-up into adulthood in order to identify the factors associated with best clinical practice including diagnostic approaches, as well as optimal treatment approaches. In this article, we comprehensively review current research on various presentations of this disease, along with current knowledge about their causes, treatments and clinical outcomes.
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