The U.S. CTEPH Registry is a multicenter, observational, U.S.-based study of the clinical
course and treatment of patients diagnosed with chronic thromboembolic pulmonary
hypertension (CTEPH), WHO Group IV Classification for Pulmonary Hypertension. The mission of
the Registry will be to promote a greater understanding of the prevalence, pathophysiology,
evaluation, and treatment of patients with CTEPH through shared information, education, and
collaborative investigation among pulmonary hypertension (PH) centers of excellence
throughout the U.S.
Chronic thromboembolic pulmonary hypertension (CTEPH) is a subset of pulmonary hypertension
(PH) characterized by the obstruction of pulmonary arteries with fibrotic material and
vascular remodeling that leads to increased pulmonary arterial pressure and right
A United States CTEPH Registry has been established under the guidance of a Steering
Committee consisting of recognized physicians and scientists with expertise in CTEPH and
related diseases. The mission of the Registry will be to promote a greater understanding of
the prevalence, pathophysiology, evaluation, and treatment of patients with CTEPH through
shared information, education, and collaborative investigation among PH centers of
excellence throughout the U.S. The development of a national CTEPH registry will be an
important element in the advancement of the understanding of CTEPH and improvement in the
care of patients who suffer from this debilitating disease.
All consecutive consenting patients recently diagnosed (< 6 months) with CTEPH at
participating study sites will be enrolled. At the time of study entry, data on prior
history and evaluation will be obtained from patient interview and review of medical
records. Patients will be followed biannually after enrollment until the conclusion of the
study (minimum of 1 year follow-up for final patients enrolled).
Much of the data gathered by the Registry will be presented as descriptive statistics. The
Registry will seek the assistance of a biostatistician to develop a statistical plan for
within patient and between patient cohort analyses.
Approximately 750 patients with newly diagnosed (< 6 months) CTEPH will be enrolled.
Participants will be followed from the time of their enrollment until 1 year after the final
patient is enrolled. Short term outcome measures and medical history will be collected
during the initial visit, and clinical data will be collected from the initial clinic
visits, hospital says, or procedures (if applicable). Patients will then be followed every 6
months after their enrollment for long term outcome measures on quality of life, functional
status, and medical treatments.
Demographics and Clinical Course:
Demographics, risk factors, time to appropriate testing and diagnosis, PAH targeted
medication use, functional status, and hemodynamics at the time of enrollment will be
provided annually as aggregate data to all investigators.
Evaluating Outcomes of PTE Surgery:
Descriptive statistics of the peri-operative data collected will be provided as aggregate
data to all investigators annually. Immediate post-operative and longitudinal follow-up of
all patients undergoing PTE surgery will also be provided as aggregate data annually to all
sites. Comparisons of hemodynamics, functional status, medication and supplemental oxygen
use and healthcare utilization will be made between the pre-operative data and longitudinal
Evaluating Predictors of Successful PTE:
Using longitudinal data collected on post-operative PTE patients, a definition of
"successful PTE" will be established based upon hemodynamics, functional status and QoL.
Retrospective analysis of pre-, peri-, and post-operative data can then be analyzed to
identify factors that predict a "successful" or "unsuccessful" PTE surgery.
Evaluating Outcomes of Nonsurgical Therapy of CTEPH:
Predominantly descriptive statistics will be used to describe why patients are not operative
candidates and the nonsurgical therapies (medication, balloon pulmonary angioplasty) used
for treatment. Changes in hemodynamics, functional status, QoL and healthcare utilization on
therapy will be provided as aggregate data annually.
Evolving Research Needs:
To adapt to the evolving science and research needs of the CTEPH community, separate
analysis plans will be developed for new objectives identified and minor changes in the eCRF
may occur in response to new diagnostics and treatments, if approved by the Steering
Committee. Ongoing assessment of enrollment and data captured will also be performed by the
Steering Committee to be certain the goals of the Registry are being met.
A Steering Committee, chaired by Dr. Kim Kerr, is comprised of thought leaders in the areas
of cardiology, pulmonology, and cardiothoracic surgery with expertise in CTEPH. The
committee will have the following responsibilities:
- Provide advice and guidance on the U.S. CTEPH Registry study design and protocol
- Provide advice and guidance on the variables to be captured by the eCRF
- Provide guidance on study sites selection
- Review data results and provide insights into interpretation of the data annually
- Provide input on the planning and organization of abstracts and manuscripts
- Review abstracts and papers for submission to scientific journals or conferences
- Present data at conferences
An Adjudication Committee, consisting of physicians with expertise in the diagnosis of
CTEPH, will review all patients felt by Investigators to be eligible for the Registry. The
adjudication committee members must confirm the diagnosis of CTEPH before the subject can be
enrolled in the Registry.
- Patients must be a permanent resident of the United States
- Documentation of the following hemodynamic parameters by right heart catheterization
- Mean pulmonary arterial pressure (PAPm) ≥ 25 mm Hg at rest and,
- Pulmonary artery wedge pressure (PAWP) ≤ 15 mm Hg (or > 15 mmHg if justified)
- Radiologic confirmation that chronic thromboembolic disease is the cause of the
pulmonary hypertension by
- One or more mismatched perfusion defect(s) by lung ventilation/perfusion scan,
- Confirmation of chronic thromboembolic disease by evidence of bands/webs, vessel
narrowing or occlusion seen on CT pulmonary angiogram (CTA), conventional
angiography or MR angiography (MRA).
- Patients must be diagnosed with CTEPH within 6 months of being considered for study
eligibility (signing of consent to participate). The date of diagnosis will be
defined as when both hemodynamic criteria have been met and chronic thromboembolic
disease is confirmed to be the cause of the pulmonary hypertension by an abnormal V/Q
scan and the presence of chronic thromboembolic disease on CTA, MRA or pulmonary
angiography. Hemodynamic and radiologic criteria can be met at separate time points;
the most recently met criteria time point will be defined as the date of diagnosis.
- Prior to enrollment all subjects must have the diagnosis of CTEPH confirmed by the
- Patients unwilling or unable to provide written consent for participation in the
study. Appropriate surrogate consent will be obtained for pediatric patients as
defined by each investigational site's IRB.
- Patients with an underlying medical disorder with an anticipated life expectancy less
than 2 years.
- Patients who do not meet inclusion criteria including:
- Have not had documentation of hemodynamic criteria by right heart
catheterization as outlined in the inclusion criteria
- Do not have radiologic confirmation of chronic thromboembolic disease as
outlined in the inclusion criteria
- Meet the criteria for inclusion into WHO Groups I, II, III, or V