Top Things to Know: Treatment Strategies for Cardiomyopathy in Children
Published: June 08, 2023
- Treatment strategies for cardiomyopathy in children should be designed to develop personalized therapy for each patient through an effort to identify a) the specific cardiac pathophysiology(ies) and, if possible, b) the root cause or specific etiology that exist in each patient.
- Pediatric cardiomyopathy should be recognized as a problem that may be diagnosed in a variety of clinical milieus: a) patients at risk for cardiomyopathy with a negative phenotype; b) patients with a positive cardiomyopathy phenotype but asymptomatic; c) symptomatic cardiomyopathy, and d) end-stage disease. Therapeutic strategies should be tailored to the specific clinical milieu in each patient.
- Heart failure in children differs in important ways from heart failure in adults. Recent studies have found distinct pathologic, transcriptomic, and proteomic profiles in pediatric heart failure compares to adults. In addition, children lack many of the comorbid conditions present in adults. These differences may influence the efficacy of adult heart failure treatments when applied in pediatric patients.
- Therapies can be initiated in some pediatric patients at risk for cardiomyopathy prior to developing a cardiomyopathy phenotype to mitigate the development or progression of disease. These therapies exist for cardiomyopathies associated with dystrophinopathies, treatment for pediatric cancer, and some metabolic disease. Clinical trials for genetic therapy in pediatric cardiomyopathy are occurring and will increase in the future.
- Pediatric clinical trials of adult heart failure therapies have demonstrated only limited evidence for their efficacy in pediatric dilated cardiomyopathy. However, some evidence exists to suggest their use has contributed to a decrease in mortality in pediatric dilated cardiomyopathy in recent experience.
- The etiologies of pediatric hypertrophic cardiomyopathy, aside from maternal diabetes, are almost all genetic with different prevalence from adult patients. An expanding number of etiology-specific therapies exist and are increasing which makes determination of etiology in pediatric hypertrophic cardiomyopathy important.
- Sudden death is a potential complication in pediatric hypertrophic cardiomyopathies with a positive phenotype. Several models have been devised to determine risk of sudden death. Therapies to prevent sudden death including exercise limitations and placement of an internal defibrillator (ICD) continue to evolve and have age and size-specific caveats compared to adult guidelines.
- Left ventricular noncompaction, restrictive, and arrhythmogenic pediatric cardiomyopathies occur less frequently than dilate and hypertrophic phenotypes but each have unique therapeutic considerations regarding thrombotic prophylaxis, heart transplantation, and exercise limitation respectively.
- Heart transplantation is an accepted therapy for end-stage pediatric cardiomyopathy. The use of ventricular assist devices as a bridge to transplantation is common in pediatrics but is associated with a low rate of ventricular recovery similar to adult experience.
- Limitations in sample size and funding in pediatric clinical trials now and in the future are being addressed with use of learning networks involving multiple pediatric cardiomyopathy centers. Innovative use of multi-center registries and adaptive trial design have the potential to advance and refine future therapy in pediatric cardiomyopathy.
Citation
Golbus JR, Lopez-Jimenez F, Barac A, Cornwell WK, Dunn P, Forman DE, Martin SS, Schorr EN, Supervia M; on behalf of the American Heart Association Exercise, Cardiac Rehabilitation and Secondary Prevention Committee of the Council on Clinical Cardiology; Council on Lifelong Congenital Heart Disease and Heart Health in the Young; Council on Quality of Care and Outcomes Research; and Council on Cardiovascular and Stroke Nursing. Digital technologies in cardiac rehabilitation: ascience advisory from the American Heart Association [published online ahead of print June 5, 2023]. Circulation. doi: 10.1161/CIR.0000000000001150