Systemic sclerosis: assessment and treatment. TIght control in a tight disease.

Systemic sclerosis(SSc) is a systemic auto immune disease, resulting in a decreased life expectancy in all, but especially in patients with diffuse cutaneous SSc. The major causes of death are pulmonary fibrosis and pulmonary hypertension. In this thesis the epidemiology of SSc and its pulmonary complications are described. Also, it offers a protocol for the diagnostic and therapeutic approach for pulmonary hypertension as complication of SSc. The prognosis of SSc could be improved if the diagnosis of SSc could be made early, in order to enable early treatment. However, to date, no diagnostic criteria for SSc exist, and the same holds true for disease activity and prognostic criteria. This thesis also describes other organ complications of SSc. First, it was found that an dilated oesophagus on a HRCT-scan of the chest, ordered in a patient to evaluated interstitial lung disease, has a high positive predictive value for the presence of SSc. Second, a description of psychological factors of importance in SSc is described. It was found that skin deformities are core stressors in SSc. Third, we described the increase of the accuracy of the echocardiography for the non-invasive diagnosis of pulmonary hypertension in SSc patients. By combining the right ventricular Tei-index, which represents the global function, with the estimated systolic pulmonary arterial pressure, the number of negative right heart catheterizations would decrease. Finally, the six-minute walking-distance (6MWT), is evaluated in four different diagnosis groups of SSc patients, divided by their pulmonary complications. We concluded that also SSc patients without any pulmonary involvement, have a decreased 6MWT compared to healthy subjects, implying decreased exercise capacity. At last, the long-term follow-up results of autologous peripheral blood stem cell transplantation in patients with severe SSc is described. After transplantation, both the survival and event free survival increased and the beneficial aspects were sustained.