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.
Innovation in pediatric medical devices is limited by financial and regulatory barriers due to the small and heterogenous target population. Intentional design of the ACTION registry has mitigated regulatory and financial barriers to pediatric VAD innovation through the use of real-world evidence gathered through a learning network.
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 We sought to develop a contemporary risk assessment tool for use in pediatric ventricular assist device (VAD) candidates to estimate risk for mortality on the device using readily available
Taking ACTION: A Prognostic Tool for Pediatric Ventricular Assist Device Mortality 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
“Inter-Rater Reliability of Total Cardiac Volume Measurement for Heart Transplantation: An Action Collaborative Study, John Dykes et al.” JOHN DYKES Download Abstract “Feasibility of Implementation of
ACTION Poster Sessions @ ISHLT Read More »
