Stem cell transplantation for rheumatic autoimmune diseases
1 Department of Rheumatology, University of Basel, Felix Platter Spital, Burgfelderstrasse 101, 4012 Basel, Switzerland
2 Musculoskeletal Research Group, Institute of Cellular Medicine, The Medical School, Framlington Place, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK
Arthritis Research & Therapy 2008, 10:217 doi:10.1186/ar2486Published: 10 October 2008
Immunoablative therapy and hematopoietic stem cell transplantation (HSCT) is an intensive treatment modality aimed at 'resetting' the dysregulated immune system of a patient with immunoablative therapy and allow outgrowth of a nonautogressive immune system from reinfused hematopoietic stem cells, either from the patient (autologous HSCT) or a healthy donor (allogeneic HSCT). HSCT has been shown to induce profound alterations of the immune system affecting B and T cells, monocytes, and natural killer and dendritic cells, resulting in elimination of autoantibody-producing plasma cells and in induction of regulatory T cells. Most of the available data have been collected through retrospective cohort analyses of autologous HSCT, case series, and translational studies in patients with refractory autoimmune diseases. Long-term and marked improvements of disease activity have been observed, notably in systemic sclerosis, systemic lupus erythematosus, and juvenile idiopathic arthritis, and treatment-related morbidity and mortality have improved due to better patient selection and modifications of transplant regimens. Treatment-related mortality has decreased to approximately 7%. Prospective, randomised, controlled clinical trials are ongoing or planned in systemic sclerosis, systemic lupus erythematosus, and several nonrheumatological conditions.