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. These 2 approaches are investigational which means that they are still being tested in research studies and are not approved by the U.S. Food and Drug Administration (FDA) for the treatment of scleroderma. If you decide to participate in SCOT, you will receive 1 of the following:

  • Stem cell transplantation: Stem cells — immature cells that can develop into different blood cells — will be withdrawn from the participant’s bloodstream. High doses of drugs to suppress the immune system will be given, followed by reintroduction of the stem cells into the blood.
  • High-dose monthly Cytoxan: Participants will receive high doses of intravenous Cytoxan, a chemotherapy drug often used to treat cancer.

About 226 people with severe scleroderma will be enrolled in North America over a 3-year period and assigned to either group. The primary objective is to evaluate differences in the rates of death and significant organ damage between the two groups 44 months after enrollment. Currently, teams of transplant physicians and rheumatologists from leading medical centers across the United States are participating in this study.

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).

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.