Project 463257

Scleroderma (or systemic sclerosis) is a rare but life-threatening disease in which the immune system attacks its own tissues, leading to excessive scarring (fibrosis) of the skin and internal organs. Scleroderma affects over 10,000 individuals in Canada and is associated with significant morbidity and mortality (with 5-year mortality estimates of up to 65%). The main cause of death in scleroderma is lung disease, with over half of patients having lung fibrosis. Scleroderma lung fibrosis is currently treated with immunosuppressive drugs (medications that calm down the immune system), with the first-line treatment being mycophenolate. Mycophenolate is usually prescribed to patients with lung fibrosis that is severe enough to cause symptoms or impact the function of the lungs, or when the disease is progressing. Unfortunately, mycophenolate does not reverse the loss in lung function once it has occurred. Recent research suggests that if we were to start immunosuppressive drugs earlier in the disease process, when lung fibrosis is detected on scans but is not yet severe enough to cause symptoms or affect lung function, then we may be able to achieve better results. We propose to conduct a pilot study to determine how best to conduct a clinical trial that would establish whether the early use of mycophenolate in mild forms of scleroderma lung disease is effective in preserving lung function. This trial will inform clinical management and has the potential of improving quality of life for people with scleroderma lung disease, by providing an opportunity to prevent severe lung disease before it is too late.