Biomarkers in Pediatric Congenital Heart Disease and PAH

NCT04130243 | Unknown

• 1 other identifier: 201700243
• Study Type: observational
• Target: 380
• Locations: 1 country
• Sites: 1

Brief Summary

Nowadays, biomarkers are broadly used in clinical practice. Blood-derived biomarkers fulfil an important role in the field of cardiology. However, most biomarkers have been investigated for adult left ventricular disease. In congenital heart diseases (CHD) and pulmonary arterial hypertension (PAH), which involves children and mostly the right ventricle, less is known about the clinical and predictive value of blood-derived biomarkers. Since the group of survivors of CHD and PAH is growing because of the improved techniques nowadays, development of better tools to maintain the quality of life for the longer term in these patients is urgently needed. Blood-derived biomarkers are minimally invasive biomarkers, are quantitative and have shown to be able to reveal pathological processes in an early stage. Hence, blood-derived biomarkers may be a good addition to current diagnostic means in CHD and PAH. Objective: The primary objective of this study is to investigate cross-sectionally the association between various emerging blood-derived biomarkers and right ventricular (RV) function:defined as tricuspid annular plane systolic excursion (TAPSE) measured with echocardiography, in children with (a history of ) an abnormally loaded, volume and/or pressure loaded, right ventricle associated with CHD and/or PAH.

Conditions

Congenital Heart Disease; Pulmonary Arterial Hypertension

Keywords

Biomarker; Congenital Heart Disease; Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (1)

• TAPSE (mm) TAPSE (tricuspid annular plane systolic excursion)

  Right ventricular function defined as TAPSE assessed by echocardiography

  At baseline (t=0)

Secondary Outcomes (11)

• WHO functional class

  At baseline (t=0)

• WHO functional class

  At 1 year

• Six-minute walk distance (6MWD)

  At baseline (t=0)

• Six-minute walk distance (6MWD)

  At 1 year

• RV fractional area change (RV-FAC)

  At 1 year

Other Outcomes (3)

• Heart/Lung transplantation per participant

  During follow-up of the study; from enrollment till end of the study (5 years later)

• Number of Hospitalizations per participant

  During follow-up of the study; from enrollment till end of the study (5 years later)

• Number of participants that do not survive during follow-up study

  During follow-up of the study; from enrollment till end of the study (5 years later)

Study Arms (2)

Congenital Heart disease

In patients with (a history of) pressure- and/or volume loaded right ventricle due to congenital heart disease extra blood will be withdrawn for a blood test; serumbiomarker

Other: Blood test

Pulmonary Arterial Hypertension

In patients with (a history of) pressure- and/or volume loaded right ventricle due to pulmonary arterial hypertension extra blood will be withdrawn for a blood test; serumbiomarker

Other: Blood test

Interventions

Blood test OTHER

Withdrawal of (extra) blood to measure serum biomarkers with a blood test

Congenital Heart disease; Pulmonary Arterial Hypertension

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population concerns all patients aged 0-18 years with (a history of) an abnormally loaded right ventricle (due to right-sided congenital heart disease or pulmonary arterial hypertension) who visit the outpatient clinic or will be electively admitted at the Center of Congenital Heart Disease - University Medical Center Groningen (CHH-UMCG).

You may qualify if:

• Age 0-18 years
• Patients with Pulmonary Arterial hypertension: diagnosis confirmed according to the standards of the National Expertise Center for PH in childhood.
• Patients with CHD with an abnormally loaded right ventricle including tetralogy of Fallot (TOF), pulmonary (valve) stenosis (PS), pulmonary insufficiency (PI), atrial septal defect (ASD), ventricular septal defect (VSD) and total anomalous pulmonary venous return (TAPVR): diagnosis confirmed by echocardiography or cardiac MRI in UMCG-CCH.

You may not qualify if:

• Age \>18 years
• Not familiar with Dutch language
• Pregnant
• Concomitant musculoskeletal disease
• No echocardiography available within three months before or after blood collection
• Being under examination for non-diagnosed disease at time of investigation

Sponsors & Collaborators

• University Medical Center Groningen: lead

Study Sites (1)

University Medical Center Groningen

Groningen, 9713GZ, Netherlands

Location

Related Publications (25)

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MeSH Terms

Conditions

Heart Defects, Congenital; Pulmonary Arterial Hypertension

Interventions

Hematologic Tests

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• A AE Verhagen, MD PhD

  University Medical Center Groningen

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 4 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Pediatric Cardiology

Study Record Dates

• First Submitted: May 1, 2017
• First Posted: October 17, 2019
• Study Start: December 5, 2017
• Primary Completion: December 1, 2023
• Study Completion: December 1, 2024
• Last Updated: October 18, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)