Hematopoietic stem cell transplantation for severe autoimmune diseases: Progress and perspectives

Two different sets of investigation are at the origin of hematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (SADs). The experimental evidence consisted in the transfer/cure of animal SADs as murine lupus by means of allogeneic but also, almost paradoxically, autologous HSCT. The clinical arm comes from serendipitous reports of patients allotransplanted for coincidental diseases, and ultimately cured of both conditions. Important multicentric prospective trials are ongoing to compare ASCT to the best available non-transplant therapies, but it may be argued that in the end both approaches will be integrated for single patients, and that new agents will possibly alter present strategies. Allogeneic STC is eliciting great expectations, but the burden of higher mortality and morbidity as a result of GVHD in the first place must be considered, even when making recourse to reduced conditioning regimens (RIC).