Two previous clinical trials suggested HSCT benefited people with severe scleroderma. ![]() ![]() “These results add to the growing evidence that stem cell transplants should be considered as a potential treatment option for people with poor-prognosis scleroderma.” Fauci, M.D., director of NIH’s National Institute of Allergy and Infectious Diseases (NIAID), which sponsored the study. “We need effective therapies for scleroderma and other severe autoimmune diseases, which can be not only debilitating to the patient but also difficult to treat,” said Anthony S. The clinical trial, called Scleroderma: Cyclophosphamide or Transplantation (SCOT), compared the safety and potential benefits of the two treatment regimens among 75 people with diffuse systemic sclerosis who had lung or kidney involvement.Ĭompared with cyclophosphamide, transplantation offered significantly greater long-term benefits, but also carried known short-term risks, such as infections and low blood cell counts. People with the disease may take antirheumatic drugs and immune-suppressing drugs like cyclophosphamide to help manage symptoms, but none of these medications has been proven to provide long-term benefit. Diffuse systemic sclerosis is a severe, often fatal form of the disease that also involves the internal organs. Scleroderma is characterized by hardening of the skin and connective tissues. 4 issue of the New England Journal of Medicine. ![]() The study, funded by the National Institutes of Health, found myeloablative HSCT to be superior to treatment with the immune-suppressing drug cyclophosphamide. The regimen, known as myeloablative autologous hematopoietic stem cell transplant (HSCT), includes chemotherapy and total body radiation to destroy the bone marrow followed by transplantation of the person’s own blood-forming stem cells to reconstitute the marrow and immune system. New clinical trial findings show that a therapeutic regimen involving transplantation of a person’s own blood-forming stem cells can improve survival and quality of life for people with severe scleroderma, a life-threatening autoimmune disease.
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