A Comparison of Morbidity Between Patients With Pulmonary Atresia With Intact Ventricular Septum and Uni- or Biventricular Circulation

Study Overview
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Brief Summary
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The goal of this observational study is to compare patients with pulmonary atresia with intact ventricular septum (PA-IVS) with univentricular circulation to patients with the same heart defect but that has a biventricular circulation in regards to mortality, quality of life, comorbidity, cardiac function, and work capacity. The main questions the study aims to answer are: • Do mortality, quality of life, comorbidity, cardiac function, and work capacity differ between patients with PA-IVS who have univentricular and biventricular circulation? Participants will be asked to answer a Quality of Life questionnaire. The investigators will also inquire with all research subjects about their participation in a series of examinations (ergo-spirometry to assess work capacity, transthoracic echocardiogram and blood sampling to assess cardiac function and cardiac health).

Official Title
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A Longterm Follow-up and Comparison of Morbidity and Heart Health Between Patients With Pulmonary Atresia With Intact Ventricular Septum and Uni- or Biventricular Circulation

Conditions
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Pulmonary Atresia With Intact Ventricular Septum

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Individuals born with pulmonary atresia with intact ventricular septum (PA-IVS) who are 15 years or older at the start of the study. Regarding the mortality variable, research subjects who have died after the age of 15 years old will be included. Exclusion Criteria: Individuals born with pulmonary atresia with intact ventricular septum who are younger than 15 years old at the start of the study.

Ages Eligible for Study
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Minimum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Biventricular circulation<br>Research subjects with PA-IVS who have biventricular circulation | |
| Univentricular circulation<br>Research subjects with PA-IVS who have univentricular circulation | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Difference in comorbidity between the two groups | Difference in myocardial infarction, heart failure, arrhythmia, stroke, malignancy, autoimmune diseases, infections, atopy, certain liver diseases, and protein-losing enteropathy between the two groups | September 2024 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Difference in quality of life between the two groups | A validated quality of life questionnaire (PROMIS Scale v1.2-Global Health) will be sent to all the research subjects and the answers will be compared between the two groups. The questionnaire consists of a ten-item patient reported measure of physical, mental and social health. Each statement is answered on a scale from 1-5, higher scores mean better outcome. | September 2024 | 
| Difference in physical capacity between the two groups | Ergospirometry, outcome variables: exercise test (exercise capacity measured in watt and over time, counted in minutes), oxygen saturation (measured in percent), blood pressure (measured in millimetre of mercury) | September 2024 | 
| Difference in cardiac capacity between the two groups | Transthoracic echocardiogram | September 2024 | 
| Difference in general health, linked to cardiac health, between the two groups | The following blood tests will be analysed: N-terminal pro-brain natriuretic peptide (a marker for heart failure), Alanine aminotransferase (a liver enzyme), Aspartate aminotransferase (a liver enzyme), Creatinine (a waste product from muscle metabolism, used to assess kidney function), Sodium (an electrolyte), Potassium (an electrolyte), Hemoglobin (a protein in red blood cells that carries oxygen), White blood cell count (Leukocyte count), Platelet count (Thrombocyte count), Albumin: A protein produced by the liver that helps maintain fluid balance in the body, TREC (T-receptor excision circles): A marker used to assess immune function, specifically the production of new T cells, Expanded lymphocyte typing (a comprehensive examination of lymphocyte subtypes) | September 2024 | 
| Difference in mortality between the groups |  | September 2024 |