Multimodality Biomarkers for Noninvasive Assessment of the Fontan Patient
Single-ventricleMyocardial FibrosisThis is a single center prospective longitudinal exercise training study and will enroll approximately 50 Fontan patients and 20 controls of a similar age, gender, BMI and physical activity level between the ages of 10-40 years. Participants will undergo an MRI of the Fontan circulation. This will include imaging of the heart, lung and liver. This will include specific imaging for tissue characterization and assessment of myocardial fibrosis, liver fibrosis and disproportionate pulmonary blood flow. The investigators will then draw blood (approximately 10 ml) for assessment of serum biomarkers and circulating microRNAs of interest. The participants will undergo exercise testing and will then start a 3-6 month long cardiac rehabilitation program. After the 3-6 month study period the participants will return back for a follow up and repeat all the testing completed at enrollement.
Fontan-Sprechstunde
Univentricular HeartAfter successful Fontan surgery, the risk of mortality in childhood is only low. Unfortunately, some of the patients suffer from Fontan-typical long-term complications in the long-term course, whereby protein loss neuropathy must be mentioned in particular, which is described in the literature with an incidence of 3-14% (1, 2) and still has a 5-year risk of death of 6-12% today (2, 3). Protein loss tereopathy leads to loss of protein in the intestine and subsequently to diarrhea and edema. Other problems concern the liver, which can develop cirrhosis due to chronic congestion (4-6). Cardiac can lead to heart failure and arrhythmias. The registry study described in this protocol is intended to identify factors that influence the treatment outcome of patients in the Fontan circulation in the long term through systematic prospective documentation of the data from our standardized and guideline-oriented treatment.
Ventripoint Single Ventricle Study
Congenital Heart DiseaseSingle-ventricleThis study will validate a coordinate-based 3-dimensional echocardiographic technique for the assessment of cardiac size and function in children and young adults with functional single ventricles.
Prospective Evaluation of Univentricular Hearts
Univentricular HeartUniventricular heart defects are among the most complex congenital malformations. The treatment of these patients usually includes 3 operations and accompanying diagnostics, some of which are invasive, within the first 3 to 4 years of life until the "fontan circulation" is established. Unfortunately, the group of patients with a univentricular cardiac malformation is also the group with the highest mortality rate until the Fontan circulation is established. The expected mortality varies depending on the complexity of the underlying intracardiac and extracardiac malformations and possible concomitant morbidity; According to data from the National Quality Assurance in Germany, it is currently up to 30% in patients with hypoplastic left heart syndrome. However, the treatment methods are well standardized and there has been a clinical protocol in Erlangen since 2008 that includes diagnostic examinations using cardiac catheterization (HKU) and magnetic resonance imaging (MRT), as well as non-invasive, instrument-based diagnostics and targeted blood tests in a consistent sequence before and after the operations regulates. The Erlangen protocol ends with the cardiac MRT six months after the Fontan operation (syn: total cavopulmonary connection [TCPC]). If MRT imaging is not possible (e.g. if a cardiac pacemaker is present) or if there is a clinical indication (e.g. an obstruction noticeable on MRT or the presence of an "overflow with right-left shunt"), HKU is performed in the first year after TCPC. These examinations are used to depict hemodynamics, cardiac function, vascular development, and lymphatic drainage disorders in order to evaluate the preoperative risk, adapt treatment strategies to the individual, and identify complications at an early stage. This concept is based on clinical experience and current scientific findings and has proven itself in everyday clinical practice since its introduction for Erlangen-Fontan patients and has proven to be very helpful for individualized treatment. After a successful Fontan operation, the mortality risk in childhood is only low. In the long term, however, some of the patients unfortunately suffer from Fontan-typical long-term complications with protein-loss enteropathy having to be mentioned in particular, which is described in the literature with an incidence of 3-14% and today still a 5-year incidence-has a mortality risk of 6-12%.
Fontan Udenafil Exercise Longitudinal Assessment Trial - 2
Single Ventricle Heart DiseaseThis study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.
Registry to Assess the Safety and Feasibility of the Subpulmonary Support With the Novel Venous...
Heart FailureUniventricular Heart3 moreThe purpose of the study is to monitor the clinical safety and performance of the EXCOR Venous Cannula in context of an EXCOR VAD therapy to ensure continued acceptability of identified risks, to enable detecting emerging risks and to assess clinical improvement on both short- and long-term.
Characterizing the Effect of Dopamine on Markers of Lymph Re-circulation in Fontan-associated Protein-losing...
DopamineProtein-Losing Enteropathies5 morePatients that have undergone a Fontan procedure (surgical correction for single ventricle congenital heart disease) may develop a complication known as protein-losing enteropathy (PLE). Some studies suggest PLE is primarily caused by impaired lymph flow. Use of continuous dopamine infusion can improve PLE. Evidence suggests the effect of dopamine may be through its effect on lymphatic function. This observational study looks at markers of lymph flow and PLE symptoms after treatment using dopamine and other standard therapies during disease exacerbations.
Simple Non-invasive Breathing Device to Improve Pulmonary Flow Pusatility in Single Ventricle Post...
Single-ventricleThis is a pilot study using a novel, minimal risk, portable, hands-free oral-positive pressure device (oPEP) in patients with Fontan palliation that will examine whether using this device in both the acute and chronic phase will alter Fontan hemodynamics and create pulsatility in the Fontan circuit and thereby increasing cardiac output. This device is easy to use and poses no significant risk to human subjects. The investigators will measure this through echocardiographic measures including pulsatility in different aspects of the Fontan circuit including IVC, hepatic veins, the Fontan conduit, and pulmonary arteries and aortic blood flow measurements. After demonstration of how to use the device appropriately, the investigators will have patients use the device after their clinical echocardiogram for their clinic appointment. The investigators will ask them to use the device at home 3-4 times a day for 10-15 mins and have them return in approximately 4 weeks to have another echocardiogram done with the same measurements.
ET1 Concentration, Metabolic Pathway Activation, and Pulmonary Blood Flow in Infants Undergoing...
Single-ventriclePulmonary Vascular Resistance Abnormality3 moreThis is a novel preliminary study of biomarkers of pathologic pre-operative pulmonary vascular development, elevated pre-operative Pulmonary Vascular Resistance Index (PVRi), and complications associated with decreased post-operative pulmonary blood flow in single ventricle patients undergoing superior cavo-pulmonary anastomosis (SCPA). The study will utilize a combined targeted and untargeted approach to both optimize translation of a promising existing biomarker and efficiently identify novel biomarkers and potential therapeutic targets in this population.
Interstage Monitoring Using an Implantable Loop Recorder: A Pilot Study
Univentricular HeartThe primary objective of this investigation is to evaluate the feasibility and safety of implantable loop recorder implantation (using the LUX-DX™) in newborns undergoing stage 1 palliation. The secondary objective of the investigation is to evaluate the feasibility of continuous measurement of heart rate and arrhythmia using an implantable loop recorder (LUX-Dx™) during the interstage period. An implantable loop recorder will be placed at the time of chest closure following stage 1 palliation (either at the time of index surgery or delayed). The device will be placed in a subclavicular pouch through the existing sternotomy incision. Participants will be in the study for 6 months to 3 years.