CirculationAHA SCIENTIFIC STATEMENTTreatment Strategies for Cardiomyopathy inChildren:A Scientific Statement From theAmerican Heart AssociationCarmel Bogle,MD;Steven D.Colan,MD;Shelley D.Miyamoto,MD,FAHA;Swati Choudhry,MD;Nathanya Baez-Hernandez,MD;Molly M.Brickler,MSN,APNP;Brian Feingold,MD,MS,FAHA;Ashwin K.Lal,MD,FAHA;Teresa M.Lee,MD;Charles E.Canter,MD,FAHA,Vice Chair;Steven E.Lipshultz,MD,FAHA,Chair,on behalf of the American Heart Association YoungHearts Pediatric Heart Failure and Transplantation Committee of the Council on Lifelong Congenital Heart Disease and HeartHealth in the Young (Young Hearts)ABSTRACT:This scientific statement from the American Heart Association focuses on treatment strategies and modalitiesfor cardiomyopathy (heart muscle disease)in children and serves as a companion scientific statement for the recentstatement on the classification and diagnosis of cardiomyopathy in children.We propose that the foundation of treatment ofpediatric cardiomyopathies is based on these principles applied as personalized therapy for children with cardiomyopathy:(1)identification of the specific cardiac pathophysiology:(2)determination of the root cause of the cardiomyopathy sothat,if applicable,cause-specific treatment can occur (precision medicine);and (3)application of therapies based onthe associated clinical milieu of the patient.These clinical milieus include patients at risk for developing cardiomyopathy(cardiomyopathy phenotype negative),asymptomatic patients with cardiomyopathy (phenotype positive),patients withsymptomatic cardiomyopathy,and patients with end-stage cardiomyopathy.This scientific statement focuses primarily on themost frequent phenotypes,dilated and hypertrophic,that occur in children.Other less frequent cardiomyopathies,includingleft ventricular noncompaction,restrictive cardiomyopathy,and arrhythmogenic cardiomyopathy,are discussed in less detail.Suggestions are based on previous clinical and investigational experience,extrapolating therapies for cardiomyopathies inadults to children and noting the problems and challenges that have arisen in this experience.These likely underscore theincreasingly apparent differences in pathogenesis and even pathophysiology in childhood cardiomyopathies compared withadult disease.These differences will likely affect the utility of some adult therapy strategies.Therefore,special emphasishas been placed on cause-specific therapies in children for prevention and attenuation of their cardiomyopathy in additionto symptomatic treatments.Current investigational strategies and treatments not in wide clinical practice,including futuredirection for investigational management strategies,trial designs,and collaborative networks,are also discussed becausethey have the potential to further refine and improve the health and outcomes of children with cardiomyopathy in the future.Key Words:AHA Scientific Statements cardiomyopathies child heart diseases precision medicineediatric cardiomyopathy is
