This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.Introduction:Systemic sclerosis (SSc) is a rare, often fatal disease with an unknown cause, which has no effective therapy. One of the most serious complications of systemic sclerosis is pulmonary hypertension (PHT), which is the most frequent cause of death from a scleroderma complication. New therapeutic advances have recently improved the short-term management of moderate to severe pulmonary hypertension in scleroderma, but the long-term outcomes are unknown. One of the major remaining questions is whether identification and treatment of patients with early pulmonary hypertension will improve outcome.Objectives:1. To understand the natural history of pulmonary hypertension in systemic sclerosis.2. To evaluate the course of disease progression in patients with systemic sclerosis and early pulmonary hypertension 3. To collect blood and cells for specific markers of PHT to test for surrogate markers of PHT and PHT progressionStudy Design:This is an observational registry study of scleroderma patients to determine the time of pathologic progression of pulmonary hypertension from pre-pulmonary hypertension, to diagnosable pulmonary hypertension, to clinical worsening of disease. Approximately 400 patients will be entered into the registry from multiple sites throughout the United States. Stanford will enroll approximately 30 patients. All patients will be followed in prospective fashion noting the clinical course of disease by both scheduled and event driven follow up for 5 years. A thorough baseline history will be collected to determine key prognostic and correlative factors for both disease prevalence and progression. Yearly follow up consisting of pulmonary function tests, echocardiogram, 6 minute walk test, and blood tests will be conducted to further understand and note the progression of scleroderma related PAH. All patients will complete bi-annual questionnaires to assess symptoms and review changes in medications. Event driven follow up will occur in the following situations: performance of right heart catheterization either for the initial diagnosis of PHT or as a follow-up to evaluate progression of disease; hospitalization; death or withdrawal of consent; or change in PHT therapy.
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