Allogeneic hematopoietic stem cell transplantation for severe autoimmune diseases

Allogeneic hematopoietic stem cell transplantation (HSCT) offers the unique opportunity to replace a diseased hematopoietic or immune system by healthy donor cells. Proof of principle comes from experimental animal data, from the experience with severe aplastic anemia, from observations of patients with concomitant autoimmune disease and from scattered case reports. Complete remissions have been described as well as failures. Allogeneic HSCT remains associated with significant morbidity and mortality. The potential risks might outweight the benefits. Currently, allogeneic HSCT cannot be recommended in general. Carefully conducted prospective studies are warranted to define target populations and best technology to be used. Conditions for such studies have been identified by an international expert panel.