Prospective Evaluation of Univentricular Hearts

Evaluation of the Standard Data Collected as Part of the Erlangen Protocol for the Treatment and Monitoring of Patients With Univentricular Heart Defects in Terms of the Fontan Procedure

Univentricular 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%.

Study Overview

• Status: Recruiting
• Conditions: Univentricular Heart
• Intervention / Treatment: Other: Univentricular Heart

Detailed Description

Univentricular 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.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Sven Dittrich, Prof.; Phone Number: 09131 85 33750; Email: sven.dittrich@uk-erlangen.de
• Study Contact Backup: Name: Isabelle Schöffl, PD; Phone Number: 09131 85 33750; Email: isabelle.schoeffl@uk-erlangen.de

Study Locations

• Germany
  • Erlangen, Germany, 91054
    • Recruiting
    • Universitätsklinikum
    • Contact: Sven Dittrich, Prof.; Phone Number: 09131 85 33750; Email: sven.dittrich@uk-erlangen.de
    • Contact: Isabelle Schöffl, PD; Phone Number: 09131 85 33750; Email: isabelle.schoeffl@uk-erlangen.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• congenital heart defect of the univentricular type and the clinical necessity of a multi-stage treatment in terms of the Fontan procedure.

Exclusion Criteria:

• no exclusion criteria

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Univentricular Heart; All patients with univentricular hearts	Other: Univentricular Heart; Prevention of complications when discovered

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of correct findings in patients after Fontan palliation to assess the outcome after TCPC	correct perioperative course after TCPC, correct anatomy after surgery without vascular or anastomotic obstructions, correct cardiovascular function, correct lymphatic vessel presentation, correct liver sonography, blood protein values in the normal range, absence of clinical symptoms	50 years
Heart or valvular insufficiency assessed by echocardiography and MRI	Measured through ejaction fraction (EF; [EF]=%) and through visual assessment of the valves with e.g. doppler echocardiography. Additionally, measurement of the same values is performed through MRI	50 years
Pathologic presentation of lymph vessels assessed by MRI	Described through various scores (Biko et al., Schröder et al.)	50 years
Hypoalbuminemia, hypoproteinemia	measured through blood analysis (in g/L)	50 years
Number of patients with Protein-losing enteropathy (PLE) or plastic bronchitis	Assessed by clinical diagnosis	50 years
Number of patients with "Failing Fontan"	Examples would be Fontan take-down, heart transplant or passing away	50 years
Prevalence and cause of early post-Fontan morbidity	assessed by clinical diagnosis	50 years

Collaborators and Investigators

• Sponsor: University of Erlangen-Nürnberg Medical School
• Investigators: Study Chair: Sven Dittrich, Prof., Universitatsklinikum Erlangen

Study record dates

Study Major Dates

• Study Start (Actual): May 20, 2021
• Primary Completion (Anticipated): May 20, 2031
• Study Completion (Anticipated): May 20, 2041

Study Registration Dates

• First Submitted: September 21, 2022
• First Submitted That Met QC Criteria: November 10, 2022
• First Posted (Actual): November 17, 2022

Study Record Updates

• Last Update Posted (Actual): November 17, 2022
• Last Update Submitted That Met QC Criteria: November 10, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Congenital Abnormalities; Heart Defects, Congenital; Cardiovascular Abnormalities; Univentricular Heart
• Other Study ID Numbers: 528_20 B

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No