The Fontan Dapagliflozin Pilot Study (FonDap)

Ari Cedars, MD - Johns Hopkins Medical Institution

Congenital heart disease is the most common form of birth defect, impacting nearly 40,000 live births each year. Medical and surgical advances have permitted the great majority of these defects to be effectively repaired in childhood. However, individuals with the most complex forms of congenital heart disease, those with only one functional pumping chamber, are an exception. Due to a lack of other options, these patients frequently end up undergoing a palliative procedure called a Fontan repair instead of a complete repair of their heart. The Fontan repair creates a very abnormal circulatory system that is prone to failing when patients are in their 30s and 40s1. Currently, there are no therapies proven to prolong survival in patients with a Fontan repair. In our study, we plan to investigate how a new treatment for Fontan patients helps improve the abnormalities in their circulatory system. For this study, we will be using a class of drugs called SGLT2 inhibitors that are very beneficial in patients with heart failure without congenital heart disease but have not been tested in patients with a Fontan repair. Because of the unique circulatory abnormalities resulting in Fontan heart failure, we believe that patients with a Fontan repair will benefit from therapy with SGLT2 inhibitors.

Improved Image Quality for Cardiac MRI in Children with Pacemakers and Defibrillators

Gregory Webster, MD, MPH - Northwestern University Feinberg School of Medicine

Cardiac magnetic resonance imaging (MRI) and cardiac implantable devices (CIEDs), such as pacemakers and implantable cardioverter defibrillators (ICDs), are both critical in congenital heart disease. CIEDs improve quality of life and can be lifesaving; they are not usually optional. Children with congenital heart disease frequently require cardiac magnetic resonance imaging (MRI). Physicians rely on MRI data to make informed decisions about clinical care and to plan surgeries. A major clinical problem is that CIEDs produce artifact in the MRI scanner, degrade the quality of the images and make clinical decisions harder. Patients need both CIEDs and high-quality MRIs. In this prospective study, our objective is to improve imaging quality for patients with CIEDs by translating existing wideband sequences (developed for adults) into pediatric care.

Application of Predictive Analytics Algorithms to Reduce Mechanical Ventilation time after Cardiac Surgery

Daniel Lee Hames - Boston Children’s Hospital

Patients with congenital heart disease (CHD) frequently require mechanical ventilation (MV) to facilitate recovery following surgery. Available MV weaning protocols do not consider the unique physiology of CHD patients. Any MV weaning protocol involving CHD must consider both the respiratory and non-respiratory support that these unique patients receive from MV. Our primary aim is to utilize three artificial intelligence (AI) based predictive analytics algorithms— each informed by continuous, high-fidelity hemodynamic and respiratory data—to develop and validate a clinical decision support system (CDSS) to guide MV weaning in CHD patients. We hypothesize that a CDSS powered by these novel risk analytic algorithms will reduce MV duration and associated morbidity in patients following surgery for CHD.

Developing a Plasma Biomarker Risk Profile for 30-Day Morbidity and Mortality for Neonates Requiring Cardiopulmonary Bypass for Congenital Heart Disease

Monique M. Gardner, MD & Nadir Yehva, MD, MSCE - Children’s Hospital of Philadelphia

Of the 1 in 100 infants in the United States born with heart disease, a quarter will require cardiac intervention in the first weeks of life. While surgical and perioperative care has advanced over the past decades, significant morbidity and mortality remains for neonates undergoing cardiopulmonary bypass (CPB) for surgical palliation of congenital heart disease (CHD).  In addition to a 30-day postoperative mortality rate of 2-9% for the most complicated operations, prolonged cardiac intensive care unit (CICU)-stay or readmissions to the CICU are also important outcomes and markers of chronic critical illness. To date, identification of high-risk infants has been limited to demographic and cardiac disease-based determinants. These clinical factors provide partial insight into the categories of patients with CHD that are at-risk but fail to address specific biologic factors contributing to poor outcomes. Additionally, clinical risk factors provide limited insight into specific mechanisms leading to poor outcomes. Blood-based biomarkers can serve as individualized, patient-based, organ-specific markers for these outcomes. Sensitive and specific early identification of at-risk patients can facilitate triaging of resources, expedite and direct more aggressive care, and delineate mechanisms leading to worse outcomes, all with the goal of improving outcomes in this vulnerable population.

Advancing development of novel, diaper worn vital sign monitor for infants with CHD

Danielle Gotlieb Sen, MS, MD, MPH - Johns Hopkins Medical Institution

Vital sign monitoring of infants outside of a traditional healthcare setting has already been shown to significantly decrease mortality for children with single ventricle congenital heart disease (1V CHD), a fragile group of patients with congenital heart disease (CHD). By monitoring oxygen saturation (SpO2), weight, hydration, intake of nutrients and diaper output, respiratory rate (RR), and temperature, studies have shown a 11-40% reduction in mortality. However, there are serious limitations in monitoring this information that has reduced the effectiveness of this intervention in real life and has prevented the expansion of remote monitoring to all infants with CHD and other fragile infant populations. In-home measurement of these vital signs requires device and application training, equipment such as a pulse oximeter, dedication, and time. It is important that the vital signs are accurately collected multiple times a day with the caregivers being relied on to collect and interpret the incoming data.

This research initiative is a continuation of the project initially funded in 2022 by The Brett Boyer Foundation and is focused on advancing progress made-to-date. 

The continued objective is to now develop an improved functional prototype and a smart phone application to further expand our clinical testing.