Early data from 500 males in the ACTION dystrophinopathy registry show substantial cardiac involvement and broad use of cardioprotective therapies. Ongoing follow-up will define the modern natural history of cardiomyopathy in dystrophinopathy.
In this multicenter retrospective study of 150 pediatric patients supported with the Impella device, 89.3% achieved positive clinical outcomes despite a 28% adverse event rate. Findings suggest a potential role for Impella in managing refractory advanced heart failure in children, particularly those with cardiomyopathy, transplant graft dysfunction, or congenital heart disease.
In a prospective study of 265 males with Duchenne Muscular Dystrophy, nearly 30% of those with moderate or severe heart dysfunction were not receiving full consensus-directed cardiac therapy, and most did not reach target medication doses—highlighting the need to optimize treatment strategies as cardiac disease becomes a leading cause of death in DMD.
Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy Read More »
The use of VADs in pediatric restrictive and hypertrophic cardiomyopathy is rare, but this ACTION multi-center registry analysis highlights that support is feasible, particularly in children with RCM. While the majority of patients were successfully bridged to transplant, outcomes were poorer in HCM patients, especially those requiring BiVAD support.
An expert opinion statement, addressing a critical gap in cardiac care for Duchenne muscular dystrophy – providing thorough recommendations for the initiation and titration of cardiac medications based on disease progression and patient response.
Abstract We report the largest pediatric multicenter experience with Impella pump use and peripheral veno-arterial extracorporeal membrane oxygenation (VA-ECMO) support. Utilizing the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) collaborative
ACTION set forth to report current outcomes in patients supported with the HeartMate 3 ventricular assist device in a multicenter learning network.
Expanding Use of the HeartMate 3 VAD in Pediatric and Adult Patients within ACTION Read More »
The results of an ACTION-wide survey of pediatric cardiology providers are detailed in the latest ACTION publication, Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD):
“Learning networks in pediatric heart failure and transplantation” was published June 17, 2021 in Pediatric Transplantation. This ACTION publication defines “learning networks” and how they’re different from traditional clinical research
Learning networks in pediatric heart failure and transplantation Read More »
