What is SCOT?
SCOT is a clinical research study designed for people with severe forms of scleroderma. SCOT stands for Scleroderma: Cyclophosphamide Or Transplantation. The SCOT study will compare the potential benefits of stem cell transplant and high-dose monthly cyclophosphamide (Cytoxan) in the treatment of scleroderma. Enrollment in SCOT was completed in May 2011. Randomization of enrolled individuals to one of the following treatments will continue through October 1, 2011.
- Stem cell transplantation: Stem cells — immature cells that can develop into different blood cells — are withdrawn from the participant's bloodstream. High doses of drugs to suppress the immune system are given, followed by reintroduction of the stem cells into the blood.
- High-dose monthly Cytoxan: Participants receive high doses of intravenous Cytoxan, a chemotherapy drug often used to treat cancer.
The primary objective is to evaluate differences in the rates of death and significant organ damage between the two groups. Currently, teams of transplant physicians and rheumatologists from leading medical centers across the United States are continuing to follow and monitor study participants.
SCOT is being sponsored by the National Institutes of Health (NIH) through its Division of Allergy, Immunology and Transplantation (DAIT) in the National Institute of Allergy and Infectious Diseases (NIAID).
Patient Experiences Video
A short video documenting the experiences of several patients undergoing stem cell treatment for scleroderma. Physician interviews providing information on the SCOT study and advances in research to treat and understand scleroderma provided.
Why is the SCOT study so important?
The SCOT study is important because more clinical evidence is needed to treat individuals with scleroderma. Currently, no treatment has been proven to prevent the disease from advancing or reverse damage to the internal organs. Since scleroderma affects individuals differently, physicians must tailor therapy to manage organ-specific symptoms. Examples of organ-specific treatments include medications such as ACE inhibitors and calcium-channel blockers, and proton-pump inhibitors. ACE inhibitors are very effective for scleroderma renal crisis. Calcium-channel blockers are useful at preventing Raynaud's attacks, and proton-pump inhibitors improve symptoms of acid reflux. Unfortunately, these medications do not affect scleroderma-associated lung, muscle, or joint disease.
While organ-specific treatment is extremely important, some experts believe that a broader and possibly more effective approach might be to treat the immune system as a whole. Stem cell transplantation and high-dose cyclophosphamide treatment are two such immune-system-based approaches. It is hoped that the results of this study will provide the information needed to define the best treatment for individuals suffering with severe scleroderma.