Chronic Thromboembolic Pulmonary Hypertension, CTEPH
Conditions
Keywords
Chronic thromboembolic pulmonary hypertension, CTEPH, Pulmonary endarterectomy, PEA, Balloon pulmonary angioplasty, BPA, Multimodal treatment
Brief summary
The goal of this observational patient registry is to learn how expert centers treat patients with chronic thromboembolic pulmonary hypertension (CTEPH). CTEPH is a condition in which blood clots block the blood vessels in the lungs. There are currently three treatment options for patients with CTEPH: * surgery to remove blood clots from large vessels in the lungs (pulmonary endarterectomy (PEA)) * the use of a small balloon to unblock smaller blood vessels (balloon pulmonary angioplasty (BPA)) * drugs Patients can also receive a combination of these treatments. The main question this registry aims to answer are: * How many patients receive a given kind of treatment? * How do expert centers combine the different treatments? * Are patients doing better after they receive a given kind of treatment? * How many patients are alive 1, 3 and 5 years after they receive a given kind of treatment? Participants will receive the same treatments that they would receive if they did not participate in the study. During the study, patients will visit their doctors as they would do normally. The doctors will collect information on the patients' health and enter it into the study database. The follow-up time will be at least 3 years for all patients.
Interventions
PROCEDUREPulmonary endarterectomy
Surgical removal of a chronic clot from the pulmonary artery
PROCEDUREBalloon pulmonary angioplasty
Catheter-based intervention to dilate occluded pulmonary vessels through the insertion and inflation of a small balloon
DRUGPulmonary hypertension (PH)-specific medication
Treatment with any PH-specific medication, i.e. endothelin receptor antagonists, phosphodiesterase type 5 inhibitors, soluble guanylate cyclase stimulators, drugs acting on the prostanoid pathway and prostaglandin I2 receptor agonists
Sponsors
Janssen Pharmaceuticals
International CTEPH Association
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Newly referred patient with CTEPH or chronic thromboembolic pulmonary disease (CTEPD) without PH according to the following criteria: 1. Mean pulmonary arterial pressure (mPAP) \> 20 mmHg at rest with pulmonary vascular resistance (PVR) \> 2 Wood units (WU); or if mPAP ≤ 20 mmHg or PVR ≤ 2 WU at rest, have exercise limitations from CTEPD without PH 2. Abnormal ventilation perfusion scan, pulmonary angiogram, computer tomographic pulmonary angiogram, or magnetic resonance pulmonary angiogram confirming CTEPD as recommended by standard guidelines 2. Treatment with anticoagulation for ≥ 3 months before diagnosis of CTEPH or CTEPD without PH 3. Naïve to interventional treatment (both PEA and BPA) 4. Pre-treatment with PH-specific medication for ≤ 12 months, or no medical pre-treatment, at enrollment 5. Willing and able to provide informed consent in order to participate in the study (informed consent signed) 6. Age ≥ 18 years 7. CTEPH-specific treatment must be modified or initiated at the participating site
Exclusion criteria
1. Main cause of PH other than CTEPH 2. Participating in an interventional clinical trial at enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Utilization and combination of treatment modalities | Min. 3 years | Proportion of patients undergoing a certain type of treatment modality, or a certain combination of modalities |
| 3-year survival | 3 years after last intervention | Proportion of patients alive 3 years after the last intervention recorded in the registry, or after start of medical treatment for patients not undergoing any interventional treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 1-year survival | 1 year after last intervention | Proportion of patients alive 1 year after the last intervention recorded in the registry, or after start of medical treatment for patients not undergoing any interventional treatment |
| 5-year survival | 5 year after last intervention | Proportion of patients alive 5 years after the last intervention recorded in the registry, or after start of medical treatment for patients not undergoing any interventional treatment |
| Changes in PH-specific medication | Min. 3 years | Proportion of patients experiencing a change in PH-specific medication during the study |
| Reason why PEA was not performed | Min. 3 years | The main reason why PEA was not performed as chosen by the investigator from a list of pre-defined reasons |
| Reason why BPA was not performed | Min. 3 years | The main reason why BPA was not performed as chosen by the investigator from a list of pre-defined reasons |
| Complications of treatment interventions (per patient) | Min. 3 years | Proportion of patients experiencing complications from BPA or PEA |
| Complications of treatment interventions (per BPA session) | Min. 3 years | Proportion of BPA sessions associated with complications |
Other
| Measure | Time frame | Description |
|---|---|---|
| Type of QoL questionnaire | Min. 3 years | Proportion of patients undergoing QoL assessment using a certain validated questionnaire during the study |
| Frequency of QoL assessment | Min. 3 years | Proportion of patients undergoing QoL assessment by means of a validated questionnaire during the study |
| Timing of unplanned hospitalizations | Min. 3 years | Analysis of the timing of unplanned hospitalization for patients experiencing an unplanned hospitalization (i.e. not considering planned hospitalizations for study procedures such as PEA or BPA) |
| Number of unplanned hospitalizations | Min. 3 years | Proportion of patients experiencing an unplanned hospitalization (i.e. not considering planned hospitalizations for study procedures such as PEA or BPA) |
Countries
Argentina, Austria, Belgium, Brazil, Canada, China, Colombia, Czechia, Denmark, Germany, India, Italy, Japan, Mexico, Poland, Singapore, Spain, Turkey (Türkiye), United Kingdom, United States
Outcome results
None listed