J Korean Rheum Assoc.  2000 Mar;7(1):13-19.

The Evolving Role of Myeloablative Chemotherapy with Stem Cell Transplantation for the Treatment of Autoimmune Disease

  • 1epartment of Internal Medicine, College of Medicine, Hanyang University Hanyang University Kuri Hospital, Kuri, Korea.


High-dose cytotoxic therapy followed by autologous stem cell transplantation has been proposed as a novel treatment modality for severe autoimmune disease. The rationale of autologous stem cell transplantation in autoimmune diseases has been based on the autoimmune animal models that marked improvement or complete eradication of autoimmune disease after syngeneic marrow transplantation. In addition, several clinical data showed that allogeneic marrow transplantation has been reported to eradicate concurrent autoimmune disease, suggesting that high-dose cytotoxic therapy may be sufficient to eradicate autoaggressive lymphocytes. Peripheral blood stem cell transplantation is widely used compared to bone marrow transplantation due to rapid marrow recovery and less treatment-related mortality. Recently, immunoablative high-dose cytotoxic therapy without stem-cell rescue also can induce complete remission in patients with refractory, severe autoimmune disease. Although several clinical data of autologous stem transplantation can achieve durable remission in severe autoimmune disease, long-term efficacy has not been fully determined yet. Further studies are needed to assess the exact role of stem cell transplantation in the treatment of severe autoimmune disease through well-designed clinical trials.


High-dose cytotoxic; Autoimmune
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