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.
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.
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.
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).
