Development and Prevention of Pulmonary Hypertension in Systemic Sclerosis

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.

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