Department of Defense Funds ACTION to Advance Duchenne Muscular Dystrophy Cardiac Research
ACTION receives $910,000 Department of Defense award to study the evolving impact of therapies on heart disease in Duchenne muscular dystrophy
Chet Villa
HeartMate 3 in Pediatric and Complex Populations: Physiologic Rationale, Patient Selection, and Management Strategies
This review outlines the physiologic principles, patient selection criteria, and specialized management strategies needed to safely use the adult-designed HeartMate 3 heart pump in children, small adults, and patients with congenital heart disease or muscular dystrophy.
Use of SGLT2 Inhibitors in Dystrophin Deficient Cardiomyopathy: A Multi-center Study Using the ACTION Registry
A new multicenter study suggests SGLT2 inhibitors — a diabetes drug class — may offer a well-tolerated way to modestly improve heart function in kids with dystrophin-deficient cardiomyopathy.
Prospective Multicenter Evaluation of a Novel Active Driving System for Pediatric Ventricular Assist Device Support
This multicenter analysis shows bivalirudin is an effective alternative to heparin for anticoagulation in pediatric mechanical circulatory support, with comparable bleeding and stroke outcomes.
Double Bridge to Heart Transplantation: Outcomes of Early vs Delayed Extra Corporeal Membrane Oxygenator Crossover in Pediatric Population
This study evaluates the progression of the distal aorta in patients with acute type A aortic dissection complicated by malperfusion syndrome who were treated with endovascular fenestration/stenting followed by delayed open aortic repair, finding that although these patients had faster distal aortic growth, their long-term survival and reoperation rates were similar to those without malperfusion syndrome.
Dystrophinopathy in ACTION: The first 500 males enrolled in the Advanced Cardiac Therapies Improving Outcomes Network prospective dystrophinopathy registry
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.
Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy
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.
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Cardiac treatment for Duchenne muscular dystrophy: consensus recommendations from the ACTION muscular dystrophy committee
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.
Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance
Partnership of cardiologists with gene transfer therapy prescribers is essential to identifing patient-specific considerations that might influence risk for adverse cardiac events.
Ventricular assist device implantation in children with a mechanical valve: An ACTION registry analysis
Patients with congenital heart disease (CHD) frequently have had valve interventions, including replacement with a mechanical valve. The impact of a mechanical valve on clinical outcomes in patients undergoing ventricular assist device (VAD) implantation is not well characterized.
