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.
Happy Fall to All! We have many exciting updates to share, such as new pediatric Impella education materials for Providers and Patients/Families, a debrief of our incredibly successful first VAD Bootcamp, and many new publications and other tools. As we move into the holiday season, we reflect on our continued appreciation and thankfulness for the engagement and collaboration from all stakeholders that continue to excel our research, quality improvement, education, and advocacy efforts.
The Pulse — Nov 2025 Read More »
A new multi-center study explores the off-label use of SGLT2 inhibitors in children and adolescents with heart failure, highlighting real-world safety data and underscoring the need for prospective trials.
Use of SGLT2 inhibitors in pediatric heart failure: a multi-center study Read More »
The iDECIDE-VAD-PEDIATRIC decision aid was developed to help families and clinicians navigate the unique challenges of pediatric ventricular assist device implantation by promoting consistent, values-based, and equitable decision-making.
Right ventricular failure (RVF) after left ventricular assist device (LVAD) implantation is a major cause of morbidity and mortality. This review offers pediatric-focused guidance on perioperative RV management, emphasizing pathophysiology, hemodynamics, and targeted interventions to optimize outcomes.
We hope you are enjoying your summer vacations and finding time to rest and rejuvenate with your families! ACTION has been busy with new publications, tools, resources, and projects (see links and features below). We are thrilled to announce the launch of our new VAD Adverse Event Definitions! These new and updated definitions will help us capture events more accurately and efficiently. The DCC also streamlined fields, improved forms, and added instructional text throughout to make data entry easier and faster. Thanks to all those who worked on this important project! Lastly, we are now taking proposals for ISHLT 2026, see more details below on the submission process.
The Pulse — Aug 2025 Read More »
This multicenter study of 367 children from the ACTION network found that 78% had a positive outcome with paracorporeal continuous flow (pCF) mechanical support. Children with congenital heart disease had lower success rates and higher mortality than those without CHD.
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 »
Through the collection and use of real-world data, the field has gained greater understanding of best practices in care of these patients, as well as improved access to devices with pediatric labeling of devices previously approved only for adults. Recent and current work across the network’s major focus areas (VAD, heart failure, muscular dystrophy, and Fontan) are highlighted.
