As 2023 brings new beginnings, ACTION reflects on what was accomplished in 2022 and what lies ahead.
This past year, we launched a first of its kind Apple watch studyfor pediatric heart failure patients. Through an App built by the ACTION team, physical functional data is being collected to better understand heart failure patients on advanced cardiac therapies. The App collects data such as heart rate, 6-minute walk tests, step count, weight, etc.
We also successfully launched our Heart Failure registry in ACTION, which will enable us to do quality improvement, research, and clinical trials for pediatric heart failure patients.
In June 2022, we were awarded a $2 million grant from Parent Project Muscular Dystrophy (PPMD) to expand muscular dystrophy focused research and quality improvement initiatives. The full press release is available here. PPMD’s gift will allow the expansion of ACTION’s initiatives including:
MD Database Expansion
Increase institution participation and ensure diverse representation
Establish use of real-world data and prospective clinical trials for treatment of cardiac disease in muscular dystrophy
Develop ACTION Patient Reported Outcomes (PRO)
Develop educational modules for patients, caregivers, and providers
We also achieved a milestone of having over 1,200 patientsentered into our Ventricular Assist Device (VAD) registry, and we launched our first prospective device trial, theBerlin Heart Active Driver Trial, using our real-world data registry model. We embarked a new education initiative for VAD patients with a 2-day series titled,In Real
Life,in which VAD providers were educated on real-life situations, scenarios and challenges.
Fontan Referral care also became a more prominent focus for ACTION in 2022. We held a quality improvement design meeting in which we identified problems, barriers, and potential interventions from our baseline data. New interventions and data collection will emerge in 2023.
Closing out 2022 academically, we are proud of the22 manuscripts we have published as a network to date. See Our Publications.
We made great strides in improving outcomes for VAD, Heart Failure, Muscular Dystrophy, and Fontan patients in 2022. As 2023 begins, we are strategically planning to develop these four areas of ACTION further. Each focus area will have a registry for data collection, quality improvement initiatives, research, education, protocol harmonization efforts, and innovation projects. We also plan to further develop our relationships with device and pharmaceutical companies and the FDA to better serve these orphan populations.
Feb 6, 2023 | 6–7 PM PHTS & Transplant Families Webinar ABO-incompatible heart transplantation – Past, present, and future Register | More Info
April 18, 2023 | 3–6 PM ACTION Spring 2023 Meeting Save the date to join us
at ISHLT in Denver, CO! More Info
Arkansas Children's Hospital Little Rock, Arkansas
Enduring Hearts 2023 Spring Direct Grant
Funding priorities for the 2023 Direct Program are:
1. to increase longevity of post heart transplant life by reducing/eliminating and the early and non-invasive detection of rejection and cardiac allograft vasculopathy (CAV)
2. evidence-based strategies to improve longevity of adolescent recipients.
Harish Gureddygari joined ACTION in December 2022 with a masters degree in computer science. He will be working as a Developer II.
Why did you want to join ACTION?
Working with the ACTION team is rewarding and gives me the satisfaction of knowing that I can make a real difference in the lives of children and their families. It also provides me with a dynamic and challenging work environment where I can grow professionally as well as personally.
What’s your favorite part about ACTION?My favorite part about ACTION is working alongside a team of talented and passionate individuals who share a common goal of improving the lives of children, and the strong culture of collaboration and teamwork that fosters in the organization.
What are some things that you enjoy doing outside of work?
Outside of work, I strive to maintain a healthy lifestyle by working out and staying active. I enjoy exploring new places and cultures, and I try to take a trip or two each year to somewhere new and exciting. I also value the time I spend with my loved ones and always try to make time for them.
Check it out!
The Berlin Heart ACTIVE Driver Trial Update
In conjunction with Berlin Heart, Inc., we launched the first prospective device trial using a pediatric ventricular assist device (VAD) registry. Historically, there has been a paucity of pediatric device and drug trials due to cost, patient volume and variability in heart failure etiology. We are collaborating with industry, patients and families and regulatory agencies to develop novel ways to bring innovative care to the bedside. We are using our registry data as ‘real world data’ for both prospective clinical trials and to apply for expanded FDA indications for pediatrics.
The Berlin Heart Active Driver study will allow young heart failure patients implanted with a Berlin Heart VAD to rehabilitate and become stronger while awaiting heart transplantation. The currently
approved IKUS driver is large, heavy and cumbersome. The new Active Driver is smaller and allows patients much greater mobility.
Carlos Bonilla Ramirez, MD: "Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Outcomes Report, Carlos Bonilla Ramirez, et al.”
Erik Frandsen, MD: "Size Isn’t Everything: Survival for Very Small Infants Supported by VAD, Erik Frandsen, et al."
Edon Rabinowitz, MD: "Post-Stage 2 Palliation Single Ventricular Assist Device Outcomes: An Advanced Cardiac Therapies Improving Outcomes Network (Action) Registry Analysis, Edon Rabinowitz, et al."
Carol Wittlieb-Weber, MD: "Taking ACTION: Creation of a Prospective Registry of Boys with Dystrophinopathy and Ventricular Dysfunction to Define Cardiac Medication Use And Optimize Guideline Directed Medical Therapy, Carol Wittlieb-Weber, et al."
Joseph Spinner, MD: "A Pediatric Heart Failure Registry is Needed: A Time for ACTION, Joseph Spinner, et al.”
Deipanjan "Deip" Nandi, MD, MSc, FAAP, FACC: "Suboptimal Titration of Heart Failure Medications in Pediatric Patients: Baseline Data from the ACTION Network, Deipanjan Nandi, et al."
Sabena Hussain, MD, MSc: "What To Do With a Squeaky Wheel? Ventricular Assist Device Use in Children With Mechanical Valves In The ACTION Database, Sabena Hussain, et al.”
Rob Niebler, MD: "Impact of Heartware VAD Discontinuation on the Pediatric Population-An Advanced Heart Failure Improving Outcomes (ACTION) Registry Analysis, Rob Niebler, et al."
Stay tuned for final presentation dates and times on our social media, as well as an ACTION schedule of ISHLT events as we get closer to the ACTION meeting at ISHLT.
Aggregate network stroke data, updated January 27, 2023.
We sustained our initial efforts, see graphs below. The leaders are working on a High-Risk bundle intervention that will be rolling out for testing soon!
Percent of patients who had a stroke with all devices (by first implant date)
Percent of patients who had a stroke with intracorporeal continuous flow device (by first implant date)
Percent of patients who had a stroke with paracorporeal continuous flow device (by first implant date)
Percent of patients who had a stroke with paracorporeal pulsatile flow devices (by first implant date)
79 HeartMate 3TM and Berlin Heart patients have been entered in the De-Escalation Project. 47 of the 79 patients achieved de-escalation before either 1. activation for transplant, 2. completing hospital course to discharge, or 3. receiving their transplant. Achieving de-escalation means the patient was extubated, off inotropes, ambulating or age equivalent, and stated feeds. This project is designed to encourage patient stability prior to transplant. It is also an effort to encourage discharge of patients on HeartMate 3TM.