Haema 2013; 4(3):275-282
by Ioanna Sakellari
Department of Haematology and Bone Marrow Transplantation, Papanikolaou General Hospital, Thessaloniki, Greece
Abstract
Autoimmune diseases (AD) are characterized by heterogeneity that finally transforms into similarity concerning the development of resistant and rapidly progressing entities that need intensive management. Novel biological treatments advance at an immense pace but do not reverse organ damage, disability or even life expectancy. During the last 15 years, serious autoimmune diseases have been treated with heavy immunosuppression and mainly autologous hematopoietic cell transplant (HCT) in order to introduce fundamental immunological changes into the structure of the immune system and the function of such naïve lymphocytes which do not promote onto autoimmunity events. Since 1997, when the preliminary results of phase I and II pioneering studies in multiple sclerosis (MS) treated with autologous HCT were published by G. Papanicolaou Hospital, great experience has been accumulated in terms of risks, benefits and economic rates of transplant as compared to biological agents. The results of both retrospective and prospective studies became the basis for randomised phase II and III studies in MS, scleroderma and Crohn’s disease. Approximately 3000 patients have undergone autologous HCT for AD worldwide and these transplants are documented in the European and International registries. The main diseases, where autologous transplant is considered a clinical option having estimated the risks and benefits of the procedure are the following: Systemic Sclerosis, Crohn’s disease, Systemic Lupus Erythematosous, Rheumatoid Arthritis, Polyomyositis, Cytopenia, Vasculitis; whereas type I diabetes mellitus and coeliac disease should be transplanted only in the context of clinical trials. There is evidence based experience concerning the peripheral blood graft (with Cytoxan plus growth factor) and the conditioning regimen. The BEAM regimen which was initially proposed by our center, is recommended for MS; while Cytoxan 200 mg/kg is generally used. The transplantation conditions must comply with the international guidelines, which require isolation during hospitalization and intensive prophylactic treatment against infections. Patient selection is the critical point for the transplant’s success and minimization of treatment-related-mortality (TRM) which ranges between 5-7% among different diseases. Selection of patients without major organ damage due to AD has resulted in the reduction of TRM over the years. Although sibling allogeneic transplant has been performed, especially in autoimmune cytopenias, it is generally avoided due to high TRM. Prospective studies and systematic registration of transplant data are strongly required in order to establish the role of autologous transplant in the management of AD.