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Blood Exchange (Plasmapheresis). Plasmapheresis is a process in which the fluid part of the blood, called plasma, is removed from blood cells. The procedure involves first withdrawing blood from the patient. The plasma, which contains the immunologically active substances, is discarded and replaced with other fluids. The blood is then returned. In a small 2001 study, this procedure appeared to slow down the course of severe progressive systemic sclerosis. Other studies are underway.

Autologous Stem-Cell Transplantation. Researchers are investigating possible benefit using transplantation of the patient's own stem cells (called an autologous transplantation). (Patients with autoimmune diseases cannot be given cells from donors.) Stem cells are the early forms for all blood cells in the body (including red, white, and immune cells). The transplant procedures introduce normal white blood cells that replace the abnormal autoimmune cells. The procedure has improved or stabilized systemic scleroderma in some patients, with remissions lasting up to 4 years. Initial results of ASTIS, a major study evaluating stem-cell transplants and high-dose immunosuppressive therapy in severe scleroderma, indicate that this combination has significant benefits with few toxic side effects. Additional research will compare stem cell transplants to monthly cyclophosphamide therapy.

There are significant risks with stem cell transplants:

  • Transplantation does not work in all patients.
  • It cannot reverse all damage that may have been done during the disease process.
  • And, importantly the treatment carries significant dangers. The mortality rate from the procedure itself is 10% in scleroderma patients, which is higher than in other patient groups who are given transplants.
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Because the procedure has serious side effects, experts suggest that the best candidates would be those at high risk for complications from scleroderma. In general, such patients would have diffuse scleroderma whose first symptoms occurred within the previous three years and who have evidence of at least mild abnormalities in the heart, lungs, or kidney. In general, patients with advanced scleroderma would not be the best candidates, because their condition is usually stable. In such cases, the risks of the procedure would outweigh the risks from the disease.

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