Review article| Volume 10, ISSUE 2, P88-96, July 1999

Treatment of severe autoimmune diseases by immunoablative chemotherapy and autologous bone marrow transplantation


      The use of bone marrow transplantation for the treatment of refractory autoimmune diseases (AD) is a new concept that is starting to emerge based on many experimental data and some clinical observations obtained in the past 10 years after either allogeneic or, more recently, autologous bone marrow transplantation. Although experimental data demonstrate that allogeneic bone marrow transplantation is effective in treating refractory AD, the treatment-related mortality of such a procedure in humans even in the absence of underlying malignancy, is such that it should only be proposed for patients with refractory AD and an underlying hematological disorder requiring bone marrow transplantation. Autologous bone marrow transplantation, or peripheral hematopoietic stem cell transplantation (HSCT), is currently performed for the treatment of various malignancies including leukemia, lymphomas, or several solid tumors, and this procedure is associated with a low, short-term mortality (<3–5%). Therefore, on the basis of both experimental and clinical data, it has recently been considered an alternative approach to treating autoimmune diseases that are refractory to conventional treatment. Since 1996, an international consensus has emerged and helped to develop some national protocols with clear inclusion criteria, especially in cases of systemic sclerosis, vasculitis, lupus erythematosus, inflammatory myositis, and rheumatoid arthritis that are refractory to conventional treatment. The aim of these phase I–II pilot studies is to define the feasibility of this new procedure, which should still be considered as experimental. Data reporting is an integral part of the protocol. For the first 145 patients reported to the Basel registry, the mortality rate associated with the procedure is 8% (similar to that for non-Hodgkin's lymphoma), well below the estimated death probability at 6 months and 5 years for most of the diseases thus far treated.


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