Clinical trial NCT01659411: Adult Congenital Heart Disease Registry (QuERI)
Official title: Adult Congenital Heart Disease Quality Enhancement Research Initiative
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pulmonary Arterial Hypertension
Keywords: Congenital Heart Disease
Pulmonary Arterial Hypertension
Lead sponsor: Actelion
Sponsor class: INDUSTRY
Collaborators: name: Canadian Heart Research Centre
First posted: 2012-08-07
Enrollment: 217
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult CHD patients meeting inclusion and exclusion criteria may be enrolled

Brief summary: Multi-center, observational, U.S.-based longitudinal program. Data will be collected prospectively for 3 years. Individual physician feedback will be provided on data collected with the purpose of improving the management of patients - quality enhancement research initiative (QuERI) process from adult patients enrolled with a history of repaired Congenital Heart Disease (CHD).

Detailed description: Approximately 800 male and female adult patients with a history of repaired CHD will be recruited from approximately 100 cardiology practices and will be followed up every twelve months for the period of three years. Consecutive patients in each practice meeting inclusion and exclusion criteria should be considered for the study. Two groups of subjects will be enrolled based on identical exclusion criteria and inclusion criteria, with the exception only of inclusion criteria #3: cohort 1- those demonstrating historic high risk criteria and cohort 2 - those demonstrating current high risk criteria.

Eligibility criteria: Inclusion Criteria: Cohort 1 (historic high risk)

1. Male and female adults (≥ 18 years of age)
2. Patients with documented history (at least one year) of an isolated, repaired congenital heart defect such as ASD, VSD, PDA, AVC (AVSD)
3. History of a large defect prior to closure as evidenced by any one of the following:

   * Size of: ASD \> 2 cm; VSD \> 1 cm; PDA \> 0.6 cm
   * Shunt 2:1 or greater
   * Pre-operative PH (PAS \> 40 mmHg) or documented shunt- related heart failure (radiographic evidence)
   * Pre-operative atrial fibrillation or flutter
4. High risk features (any one the following):

   * Age \> 40 years
   * Later surgical repair:

     i. ≥ 2 years of age for PDA or VSD ii. ≥ 1 year of age for AVC iii. ≥ 10 years of age for ASD
   * Sinus venosus defect
   * Primum defect
   * WHO functional class \> 1
   * Atrial fibrillation or flutter
5. Echocardiographic evidence of high risk features. Any one of the following:

   * Degree of TR that is mild or greater
   * Right ventricular (RV) systolic dysfunction
   * Evidence of RV dilatation: Any one of the following:

     i. Evidence of RV dilation by general, qualitative assessment ii. RV end diastolic diameter \> 3.2 cm on apical 4 chamber view at the tips of the tricuspid valve iii. RV to LV ratio at end-diastole \> 0.6 (RV and LV end diastolic dimensions measured from apical 4 chamber view, 1 cm apical of the respective valve annuli)
   * Any abnormality in the motion of the inter-ventricular septum
6. Ability and desire to execute the consent for follow up

Inclusion Criteria: Cohort 2 (current high risk)

1. Male and female adults (≥ 18 years of age)
2. Patients with documented history (at least one year) of an isolated, repaired congenital heart defect such as ASD, VSD, PDA, AVC (AVSD)
3. Current (within the last 12 months) evidence of 1 or more of the 7 following criteria:

   * Desaturation on exercise (92% or less)
   * 6 MWD \<380 m
   * PFT demonstrating DLC \<70% predicted \& FEV1\>70% predicted
   * ECG demonstrating i) RAD and ii) RVH or RAE
   * Physical findings of edema accompanied by elevated JVP and +HJR
   * CXR evidence of enlarged main and/or hilar pulmonary arterial shadows in association with right ventricular enlargement
   * Elevated biomarks (BNP or NT-proBNP above upper limit of normal)
4. High risk features (any one of the following:)

   * Age \> 40 years
   * Later surgical repair:

     i. ≥ 2 years of age for PDA or VSD ii. ≥ 1 year of age for AVC iii. ≥ 10 years of age for ASD
   * Sinus venosus defect
   * Primum defect
   * WHO functional class \> 1
   * Atrial fibrillation or flutter
5. Echocardiographic evidence of high risk features. Any one of the following:

   * Degree of TR that is mild or greater
   * Right ventricular (RV) systolic dysfunction
   * Evidence of RV dilatation: Any one of the following:

     i. Evidence of RV dilation by general, qualitative assessment ii. RV end diastolic diameter \> 3.2 cm on apical 4 chamber view at the tips of the tricuspid valve iii. RV to LV ratio at end-diastole \> 0.6 (RV and LV end diastolic dimensions measured from apical 4 chamber view, 1 cm apical of the respective valve annuli)
   * Any abnormality in the motion of the inter-ventricular septum
6. Ability and desire to execute the consent for follow up

Exclusion Criteria:

1. Poor mental function, drug or substance (e.g., alcohol) abuse, or unstable psychiatric illness, which, in the opinion of the investigator, may interfere with optimal participation in the study
2. Diagnosis of PAH (defined as RHC demonstrating mPAP ≥ 25 mm Hg and PCWP ≤ 15 and PVR \> 3 WU or PVR (indexed) \> 4 WU or treatment with PAH specific therapy) after surgical repair and prior to visit 1
3. Prior inclusion in this registry

Primary outcomes:
measure: To characterize the clinical course in a cohort of adult patients with repaired CHD at risk for developing PAH
timeFrame: screening (visit 1) through end of study (3 years)
Secondary outcomes:
measure: To characterize the clinical outcomes in a cohort of adult patients with repaired CHD at risk for developing PAH
timeFrame: screening (visit 1) through end of study (3 years)
Other outcomes:
Locations:
Study officials:
affiliation: Harvard Medical School / Boston Adult Congenital Heart
name: Michael Landzberg, MD
role: STUDY_CHAIR
References: