Project 172094

Scleroderma (SSc) is a disease that can affect the skin, joints and internal organs (heart, lungs, kidneys). There is no cure. The leading cause of death for Canadians with SSc is high pressure in the lungs called pulmonary arterial hypertension (PAH). Some experts suggest use of a blood thinner called warfarin for patients with PAH. A major side effect of warfarin is that it can cause severe bleeding. Whether this drug improves survival is not known. Using a novel research approach, this series of 3 studies will evaluate if warfarin improves survival in SSc-PAH and idiopathic-PAH (I-PAH). Part 1 will study if warfarin improves survival by comparing the survival of SSc-PAH and I-PAH patients who did and did not take warfarin. Part 2 will study specialists' beliefs about the benefits/risks of warfarin to determine if there is enough evidence to affect patient care. Part 3, a decision analysis, will inform patients and doctors about how the risks/benefits of warfarin compare with newer, more expensive treatment options. This program of research will be the first to study if warfarin improves survival in this deadly disease, and will develop innovative research methods for all researchers who study rare diseases.