Treatment Strategies for Cardiomyopathy in Children: Key Points

Bogle C, Colan SD, Miyamoto SD, et al., on behalf of the American Heart Association Young Hearts Pediatric Heart Failure and Transplantation Committee of the Council on Lifelong Congenital Heart Disease and Heart Health in the Young (Young Hearts).
Treatment Strategies for Cardiomyopathy in Children: A Scientific Statement From the American Heart Association. Circulation 2023;Jun 8:[Epub ahead of print].

The following are key points to remember from an American Heart Association Scientific Statement on treatment strategies for cardiomyopathy in children:

  1. A 4-stage system has been proposed for therapeutic interventions in heart failure (HF):
    • Stage A (at risk patients): The patient is at risk for HF but has no structural heart disease or symptoms of HF.
    • Stage B (asymptomatic patients): Structural heart disease but without signs or symptoms of HF.
    • Stage C (symptomatic patients): Cardiomyopathic heart disease with current or past symptoms of HF.
    • Stage D (refractory patients): Refractory HF requiring specialized interventions.
  2. While extrapolating results of studies from adults may be reasonable in certain diseases or age groups, it is not uniformly appropriate.
  3. Patients with muscular dystrophies are often stage A and at risk of HF. Current treatment guidelines recommend initiation of angiotensin-converting enzyme (ACE) inhibition therapy in adolescence. Novel therapies such as single exon skipping, adeno-associated virus gene therapy, and gene-editing techniques hold promise in the treatment of patients with dystrophinopathies.
  4. Current evidence does not support using standard oral HF therapies to prevent treatment-related cardiotoxicity in asymptomatic children who have received cardiotoxic cancer therapies.
  5. Cardioprotective medications such as the iron chelator dexrazoxane may prevent or reduce anthracycline-related cardiotoxicity when given concurrently with chemotherapy agents.
  6. In patients with a first-degree relative with dilated cardiomyopathy (DCM), annual screening is recommended for children 0-5 years of age, every 1-2 years for children 6-12 years of age, every 2-3 years for children 13-19 years of age, every 2-3 years for adults 20-50 years of age, and every 5 years for adults >50 years of age.
  7. A recent study showed improvement in left ventricular ejection fraction and reduced natriuretic peptide concentrations in children receiving ivabradine. The majority of children enrolled in the study were on ACE inhibitors or angiotensin-receptor blockers, mineralocorticoid receptor antagonists, and beta blockade.
  8. For pediatric patients with hypertrophic cardiomyopathy, cause-specific diagnosis is increasingly important given the increasing availability of disease-specific therapies.
  9. Pulmonary artery banding has emerged as a potential therapy for infants with DCM and preserved ventricular function. While some studies have shown this as a therapy to delay or avoid heart transplantation, further prospective study is required.
  10. Collaborative networks and registries, including ACTION (Advanced Cardiac Therapies Improving Outcomes Network), will have an important role in understanding the natural history, enhancing quality of care, and providing platforms for research of these relatively rare disease processes.

Clinical Topics: Cardiac Surgery, Cardio-Oncology, Congenital Heart Disease and Pediatric Cardiology, Heart Failure and Cardiomyopathies, Invasive Cardiovascular Angiography and Intervention, Prevention, Aortic Surgery, Cardiac Surgery and Arrhythmias, Cardiac Surgery and CHD and Pediatrics, Cardiac Surgery and Heart Failure, Congenital Heart Disease, CHD and Pediatrics and Arrhythmias, CHD and Pediatrics and Interventions, CHD and Pediatrics and Prevention, Acute Heart Failure, Heart Transplant, Interventions and Structural Heart Disease

Keywords: Angiotensins, Anthracyclines, Cardiomyopathies, Cardiomyopathy, Dilated, Cardiomyopathy, Hypertrophic, Cardiotoxicity, Chelating Agents, Dexrazoxane, Genetic Therapy, Heart Defects, Congenital, Heart Failure, Heart Transplantation, Ivabradine, Mineralocorticoid Receptor Antagonists, Natriuretic Peptides, Neoplasms, Pediatrics, Secondary Prevention, Stroke Volume, Ventricular Function, Left

< Back to Listings