The PAtients pResenTing With COngenital HeaRt DIseAse Register (ARTORIA-R)

Study Overview
=================
Brief Summary
-----------------
Advances in surgical and medical care have led to improved outcomes in patients with congenital heart disease (CHD). As a consequence, the majority of patients nowadays survives to adulthood (adults with CHD, that is, adult CHD [ACHD]) with good quality of life. Despite the surgical success, the morbidity and mortality of ACHD is higher than in the general population and is linked to the development of heart failure (HF) in adulthood. HF occurs in approximately 25% of patients with ACHD, even in those patients in whom the congenital mal-formation has been corrected successfully in childhood. The time course and presentation are heterogeneous owing to variable congenital malformation and limitation of treatment options. ACHD with an anatomic right ventricle as the systemic ventricle (e.g., atrial switch operation in patients with transposition of the great arteries [TGAs]) and those with a functional single ventricle (e.g., Fontan circulation) appear to be at higher risk of developing HF. Young age at initial corrective surgery-often in the ﬁrst 2 years of life-and lack of speciﬁc medical therapies can contribute to a high and early demand for heart transplantation in patients with ACHD.

Detailed Description
-----------------
The ARTORIA-R is an international, observational research project collecting retrospective data from 17 countries in the period 1989 to 2020 and will thereafter include data prospectively (Figure 1). These data include patient profiles at time of evaluation for listing, listing, waiting list dynamics, post-transplant data and basic donor data. Future data will be updated annually in July each year and each institution willing to contribute anonymised data to the register is invited to participate. Furthermore, the institutions and organ allocation agencies have to obtain a positive vote of the ethic committee responsible for the data transmission of anonymized patient data. The inclusion criteria are: a. The patient has to be listed as an adult transplant candidate in the country the data is obtained with an age ≥18 years b. The patient has to have a congenital heart defect or an inherited cardiomyopathy (specific; hypertrophic cardiomyopathy, arrhythmogenic right ventricular cardiomyopathy or non-compaction cardiomyopathy) which is often included into the category ACHD c. Data is obtained from the first evaluation for listing or listing for heart-only or heart-combined organ transplantation d. Transfer of anonymised data e. The institution/organization agrees to the memorandum how data is managed, and scientific cooperation is planned between all institutions. The registry has the aim to achieve meticulous information regarding the underlying congenital heart defect and the previous treatment of the patient. With these information the patients can be divided into different cohorts with a systemic left ventricle, a systemic right ventricle or a single ventricle (which can be either an anatomic left or right ventricle). Further data is acquired regarding the medical treatment, the haemodynamic evaluation of the patient, laboratory testing to assess additional organ function of the kidney or liver, treatment in the intermediate care or intensive care unit. As it is of special relevance in ACHD patient treatment of arrhythmia, antiarrhythmic medication and use of ICD or cardiac resynchronization therapy are evaluated. Were available the data regarding imaging at listing with ejection fraction for the systemic ventricle with echocardiography or cardiac magnetic resonance imaging are obtained. The exclusion criteria is: a. The patient is listed for a second heart transplantation (retransplantation) During the ongoing study additional institutions and organizations will be asked to include their data as well. Although the initial data is retrospective additional data into the register will be entered prospectively.

Official Title
-----------------
The PAtients pResenTing With COngenital HeaRt DIseAse Register (ARTORIA-R): A Global Register to Investigate Factors Associated With Morbidity and Mortality in Adult Patients With Congenital Heart Disease (ACHD) on the Waiting List for Heart or Heart/Lung Transplantation

Conditions
-----------------
Congenital Heart Disease, Heart Failure, Transplant; Complication, Failure, Arrythmia, Ventricular Dysfunction

Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: The patient has to be listed as an adult transplant candidate in the country the data is obtained with an age ≥18 years The patient has to have a congenital heart defect or an inherited cardiomyopathy (specific; hypertrophic cardiomyopathy, arrhythmogenic right ventricular cardiomyopathy or non-compaction cardiomyopathy) which is often included into the category ACHD Data is obtained from the first evaluation for listing or listing for heart-only or heart-combined organ transplantation Transfer of anonymised data The institution/organization agrees to the memorandum how data is managed, and scientific cooperation is planned between all institutions Exclusion Criteria: a. The patient is listed for a second heart transplantation (retransplantation)

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patient transplanted due to ACHD and heart failure<br>Patients successfully transplanted due to congenital heart failure. | |
| ACHD listed due to heart failure for heart or heart and combined organ transplantation<br>Patients on the actual waiting list for heart or heart and combined organ transplantation. They can have either outcome transplantation or the primary outcome death on the waiting list or delisting due to clinical worsening. The secondary outcome is as well delisting due to clinical improvement. | |
| ACHD evaluated for heart transplantation<br>All patients evaluated for heart or heart and combined organ transplantation at the hospital level | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Delisting due to clinical worsening or death on the waiting list | If the patients has a clinical worsening of their status due to advanced heart failure he or she is removed from the waiting list. Patients can as well die while on the waiting list as a consquence of heart failure | The primary outcome will be assessed at entering data into the study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Delisting due to clinical improvement | Patients can improve from the status at listing and can be removed from the waiting list due to clinical improvement of their clinical status | The secondary outcome will be assessed at entering data into the study |