Clinical trial NCT01840748: Development and Prevention of Pulmonary Hypertension in Systemic Sclerosis
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Systemic Sclerosis
Pulmonary Hypertension
Lead sponsor: University of Paris 5 - Rene Descartes
Sponsor class: OTHER
Collaborators: name: European Union
name: University of Giessen
name: University of Zurich
name: University of Florence
name: University of Campania Luigi Vanvitelli
name: University of Basel
name: University College, London
name: Charite University, Berlin, Germany
name: University of Pecs
name: University of Leeds
name: Schoen Klinik Hamburg Eilbek
First posted: 2013-04-26
Enrollment: 960
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population are adult and juvenile systemic sclerosis patients from the EUSTAR cohort (MEDSonline database) and the juvenile systemic sclerosis working group cohort

Brief summary: Systemic sclerosis (SSc) is an orphan, multiorgan disease affecting the connective tissue of the skin and several internal organs.

Pulmonary hypertension (PH) is a fatal disorder characterized by an increase in pulmonary vascular resistance, which leads to right ventricular failure. Despite being recently the object of greater attention and despite therapeutic advances, pulmonary hypertension due to SSc remains associated with a dismal 47 - 67% 3-year survival. Among SSc patients prospectively followed in the "European League Against Rheumatism Scleroderma Trials and Research" (EUSTAR) cohort, 26% of death was related to pulmonary hypertension. Although some previous data have suggested the protective effects of calcium channel blockers on the development of pulmonary hypertension, the potential preventive effects of vasodilators for the prevention of Pulmonary hypertension have not been determined yet. In addition to be considered routinely for the treatment of SSc-related pulmonary hypertension, prostanoids, endothelin receptor antagonists (ETRA) and Phosphodiesterase-5 inhibitors (PDE5i) can also be used for this indication.

This observational trial is one out of five observational trials of the collaborative project "To decipher the optimal management of systemic sclerosis" (DeSScipher).

Aim of this observational trial is:

- to compare the outcomes of adult and juvenile SSc patients who are at high risk of developing pulmonary hypertension and are receiving either different vasodilator treatments or no vasodilator treatment.


Eligibility criteria: Inclusion Criteria:

* Juvenile and adult Systemic sclerosis patients, with diagnosis according to the ACR/EULAR adult SSc criteria and PRES/ACR/EULAR juvenile SSc criteria respectively
* Patients at high risk of pulmonary hypertension with a Cochin Risk prediction score \>/= 3

ACR = American College of Rheumatology; EULAR = European League Against Rheumatism; PRES = Pediatric Rheumatology European Society

Primary outcomes:
measure: The number of patients with pulmonary hypertension at 2 years
timeFrame: 2 years
Secondary outcomes:
measure: Time to development of precapillary pulmonary hypertension
timeFrame: 2 years
Other outcomes:
Locations:
city: Paris
country: France
facility: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016
city: Bad Nauheim
country: Germany
facility: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology
city: Berlin
country: Germany
facility: Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie
city: Hamburg
country: Germany
facility: Centre for Pediatric Rheumatology, Klinikum Eilbek
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem - University of Pecs
city: Firenze
country: Italy
facility: University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine
city: Napoli-Italia
country: Italy
facility: Policlinico, Via Pansini
city: Basel
country: Switzerland
facility: Felix-Platter Spital, University of Basel
city: Zurich
country: Switzerland
facility: University of Zurich, Department of Rheumatology
city: Leeds
country: United Kingdom
facility: The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital, University College London
Study officials:
affiliation: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016
name: Yannick Allanore, Prof.
role: PRINCIPAL_INVESTIGATOR
affiliation: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology
name: Ulf Müller-Ladner, Prof.
role: STUDY_CHAIR
References: