Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
- Conditions
- Hypertension, Pulmonary
- Registration Number
- NCT02610660
- Lead Sponsor
- Association for Pediatric Pulmonary Hypertension
- Brief Summary
The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood.
Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables that have been proposed as treatment goals in PePH and the reasons for changes in treatment strategy.
The TOPP-2 registry uses the new clinical classification of PH as outlined at the 5th World Symposium for Pulmonary Hypertension (WSPH) in Nice 2013 and includes new characterizations for children with PH.
The registry is planned and implemented under the scientific leadership of the Association for Pediatric Pulmonary Hypertension (PePH), independently from the financial sponsors.
All enrolled patients will have a follow-up period of 18 months.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 388
-
Patient must be an incident patient, i.e.newly diagnosed with PH
-
Age at time of diagnosis is at least 3 months and less than 18 years
-
Patients must present with PH belonging to one of the following categories
- Group 1 according to updated Nice clinical classification
- Group 3 according to updated Nice clinical classification
- Group 4 according to updated Nice clinical classification
- Group 5 according to updated Nice clinical classification
-
PH confirmed by heart catheterisation (HC)
-
At HC, the patient must present with mean pulmonary arterial pressure (PAP) of at least 25 mmHg at rest, a pulmonary vascular resistance index (PVRi) equal to or below 3 Wood units*m^2 and mean pulmonary arterial wedge pressure (PAWP) below or equal to 15 mmHg
-
In case of congenital heart disease (CHD) patients who had undergone palliative procedure or repair to close systemic to pulmonary shunt, the diagnosis of PH must have been confirmed by HC at least 6 months after surgery/palliative procedure took place
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For patients with PAH-CHD open shunt, only those considered not operable due to advanced pulmonary vascular disease are eligible
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Patients to be included into the registry, and/or their legal guardians, must give informed consent. Where applicable patients will be asked for their written assent
-
Participating sites must agree to adhere to the recommendations of the WSPH 2015 Nice, Pediatric Taskforce.
- Patients belonging to Group 2 according to updated Nice clinical classification
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Transplantation Over registry run-time (5.5 years) Death Over registry run-time (5.5 years) Adverse events Over registry run-time (5.5 years)
- Secondary Outcome Measures
Name Time Method Type of treatment Over registry run-time (5.5 years) Type of treatment (drug, mono/combination, administration route)
Worsening of echocardiographic parameters (ECHO) Over registry run-time (5.5 years) Parameters encompass TAPSE and RV/LV dimension ratio
Increase in Brain Natriuretic Peptide (BNP) Over registry run-time (5.5 years) Escalation of treatment Over registry run-time (5.5 years) Hospitalisation related to pulmonary arterial hypertension (PAH) Over registry run-time (5.5 years) Potts shunt Over registry run-time (5.5 years) Switch in treatment Over registry run-time (5.5 years) Increase in N-terminal-proBNP (NT-proBNP) Over registry run-time (5.5 years) Use/initiation of i.v./s.c. prostanoids Over registry run-time (5.5 years) Time to clinical worsening Over registry run-time (5.5 years) Various composites of above parameters
Decline in 6-minute walk test (6MWT) Over registry run-time (5.5 years) Decline in Panama functional class Over registry run-time (5.5 years) Atrial septostomy Over registry run-time (5.5 years) Reasons for treatment change Over registry run-time (5.5 years) Decline in WHO functional class Over registry run-time (5.5 years)
Trial Locations
- Locations (32)
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
National Heart Institute
🇲🇽Mexico City, Mexico
University Children's Hospital
🇨ðŸ‡Zurich, Switzerland
Hôpital des Enfants
🇨ðŸ‡Geneva, Switzerland
Hospital Pediátrico La Misericordia
🇨🇴Bogota, Colombia
Keio University
🇯🇵Tokyo, Japan
Stanford Children's Hospital
🇺🇸Palo Alto, California, United States
University of Colorado Denver School of Medicine, Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
St Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Vanderbilt University Children's Hospital
🇺🇸Nashville, Tennessee, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Royal Children's Hospital
🇦🇺Melbourne, Australia
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
University of São Paulo
🇧🇷São Paulo, Brazil
Fu Wai Hospital
🇨🇳Beijing, China
University Children's Hospital Ulm
🇩🇪Ulm, Germany
Institute of Cardiology
ðŸ‡ðŸ‡ºBudapest, Hungary
Université Paris Descartes
🇫🇷Paris, France
University of Padova
🇮🇹Padova, Italy
Toho Universit Omori Medical Center
🇯🇵Tokyo, Japan
Beatrix Children's Hospital, University Medical Center Groningen
🇳🇱Groningen, Netherlands
Hadassah, Hebrew University Medical Center
🇮🇱Jerusalem, Israel
The Queen Silvia's Children's Hospital
🇸🇪Gothenburg, Sweden
Children's Memorial
🇵🇱Warsaw, Poland
King Fahd Armed Forces Hospital
🇸🇦Jeddah, Saudi Arabia