Autologous hematopoietic stem cell transplantation (AHSCT) for severe and refractory autoimmune diseases has been performed since 1996 with encouraging results. In general, two thirds of the patients achieve durable remissions, although morbidity and mortality related to transplantation or to relapse and progression of autoimmune diseases are still significant. Based on those results and on beneficial effects of moderate immunosuppression on the course of type I insulin-dependent diabetes mellitus (DM-I), we started in December 2003 a clinical protocol of AHSCT for this disease, in cooperation with the Northwestern University, Chicago, University of Miami and the National Institutes of Health. Under 35-year-old patients with <6 weeks from diagnosis or at the asymptomatic phase ("honey moon") of the disease have their hematopoietic stem cells mobilized with cyclophosphamide (2 g/m²) and G-CSF, collected from peripheral blood collected and cryopreserved. After conditioning with cyclophosphamide (200 mg/kg) and rabbit anti-thymocyte globulin (4.5 mg/kg) and infusion of AHSC, the patients are followed for 5 years in regards to clinical, endocrinological and immunological aspects of the disease. This clinical study may represent an important contribution of the Brazilian bone marrow transplantation community to the modern era of cell therapy for inflammatory and degenerative diseases.
Insulin-dependent diabetes mellitus; hematopoietic stem cell transplantation; autologous transplantation; autoimmune diseases