Pulmonary arterial hypertension (PAH) is the leading cause of mortality in patients with scleroderma. With a prevalence of scleroderma of 240 patients/million, and a conservative estimate that 10% of patients will develop PAH, scleroderma-related PAH (PAH-SSc) is probably the leading cause of pulmonary hypertension within the category of PAH-related diseases. Therapeutic trials indicate that PAH-SSc has a significantly poorer response to therapy compared with idiopathic PAH. A limited progress in diagnosis and management of PAH-SSc includes lack of specific outcome measures (that factor in the components of the cardiovascular response) and limited knowledge on the phenotypic, genomic and genetic characteristics that predict the development of PAH and the response to therapy and survival.
The focus of this seminar was to better understand:
- the vasculopathy characteristics in scleroderma that lead to PAH
- the value of outcome measures reliable in scleroderma
- current and future targeted therapy for this devastating disease.