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 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.
This study surveyed pediatric heart failure practitioners across ACTION centers to understand thromboprophylaxis practices in children with cardiomyopathy. The survey explored decision-making factors and medication choices using three clinical vignettes: dilated cardiomyopathy (DCM), left ventricular non-compaction (LVNC), and restrictive cardiomyopathy (RCM).
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.
ACTION and the Academic Research Consortium (ARC) aimed to harmonize definitions of pediatric and CHD AEs for use in clinical trials, registries, and regulatory evaluation.
Patients with Fontan failure are high-risk candidates for heart transplantation and other advanced therapies. Understanding the outcomes following initial heart failure consultation can help define appropriate timing of referral for advanced heart failure care.
Outcomes after initial heart failure consultation in Fontan patients Read More »
Abstract Advanced Cardiac Therapies Improving Outcomes Network (ACTION) and Pediatric Heart Transplant Society (PHTS) convened a working group at the beginning of 2020 during the COVID-19 pandemic, with the aim
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 »
Purpose Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in
