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

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

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Pulmonary Atresia With Intact Ventricular Septum

• Study Type: Observational
• Enrollment (Estimated): 140

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population consists of all individuals with PA-IVS who have been registered in Swedcon (a national register for patients with congenital heart defects) or in the National Patient Register and Cause of Death Register since 1980 and meet other inclusion criteria, including age.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Biventricular circulation; Research subjects with PA-IVS who have biventricular circulation
Univentricular circulation; Research subjects with PA-IVS who have univentricular circulation

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Vastra Gotaland Region

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2023
• Primary Completion (Estimated): December 1, 2023
• Study Completion (Estimated): September 1, 2024

Study Registration Dates

• First Submitted: June 4, 2023
• First Submitted That Met QC Criteria: June 22, 2023
• First Posted (Actual): July 3, 2023

Study Record Updates

• Last Update Posted (Actual): August 23, 2023
• Last Update Submitted That Met QC Criteria: August 22, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Congenital Abnormalities; Heart Valve Diseases; Cardiovascular Abnormalities; Vascular Malformations; Heart Defects, Congenital; Pulmonary Atresia
• Other Study ID Numbers: PA-IVS, 2023

Drug and device information, study documents

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