Summary
Pediatric heart failure (HF), though less common than adult HF, is associated with higher mortality, intensive resource use, and frequent need for advanced therapies, particularly among children with congenital heart disease (CHD). However, pediatric HF care has historically been limited by small, diverse patient populations and a lack of targeted therapies.
To address these challenges, the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) was founded in 2017. It is a global learning health system uniting 76 centers and multiple partners to improve pediatric and congenital HF outcomes through real-world data collection, quality improvement, and collaboration with stakeholders including the FDA and industry.
Key ACTION Initiatives:
Ventricular Assist Devices (ACTION-VAD):
- Focus on reducing strokes in children on VAD support through anticoagulation protocols, blood pressure control, and education.
- Stroke rates have decreased from ~29% to 11% following interventions.
- Ongoing initiatives include the MARCH study evaluating aspirin-free regimens in children with HeartMate 3 devices.
Heart Failure (ACTION-HF):
- Launched a registry to track HF admissions, treatments, and outcomes.
- Developed consensus medication protocols to reduce variability and improve titration of HF therapies.
- Investigates the use of technology like wearables and CardioMEMs to monitor patients outside the hospital.
Muscular Dystrophy (ACTION-MD):
- A registry launched in collaboration with Parent Project Muscular Dystrophy tracks over 1,000 patients.
- Developed harmonized treatment guidelines and gene therapy management protocols for Duchenne and Becker muscular dystrophies.
Fontan Patients (ACTION-FON):
- Focused on improving outcomes in single-ventricle CHD patients, especially around timely referrals and VAD management.
- Created referral guidelines and harmonization protocols for this unique population.
Industry and FDA Collaboration:
ACTION’s real-world data has helped expand FDA labeling of devices like HeartMate 3 and Impella VADs for pediatric use and is supporting new device trials, such as the EXCOR Active mobile unit.
Conclusion:
ACTION has made substantial progress in improving care, expanding device access, and fostering research for pediatric HF patients. It continues to grow globally and adaptively address emerging challenges through its learning health system model.
This ACTION study was available online in JHLT Open on June 1, 2025. Congratulations to the authors:
- Lydia K. Wright, MD, MSc,
- David M. Peng, MD
- Joseph A. Spinner, MD
- Jenna M. Murray, NP
- Matthew J. O’Connor, MD
- Christina J. VanderPluym, MD
- David N. Rosenthal, MD
- Lauren Smyth, MHA
- Angela Lorts, MD, MBA
CITATION
Wright, Lydia K. et al., Improving pediatric heart failure outcomes through collaboration and innovation: the current status of the Advanced Cardiac Therapies Improving Outcomes Network (ACTION), JHLT Open, Volume 0, Issue 0, 100311; DOI: 10.1016/j.jhlto.2025.100311
