Book Description
Autologous hematopoietic stem cell transplantation (AHSCT) was proposed 20 years ago as an alternative therapeutic approach for patients with severe and refractory autoimmune diseases. Since then, this innovative therapy has been successfully used to treat several autoimmune diseases, such as neurological disorders (Multiple Sclerosis), connective tissue diseases (Systemic Sclerosis and Systemic Lupus Erythematous), gastrointestinal inflammatory diseases (Crohn’s Disease) and others (Juvenile Arthritis, Type 1 Diabetes, Vasculitis). Experience in phase I-II and III clinical trials over the years has led to increased safety and efficacy of this procedure. Recent studies have demonstrated superior therapeutic efficacy of AHSCT versus conventional therapies, showing that transplantation can induce long-term disease remission in the absence of any further immunosuppressive treatment. Immune monitoring studies have showed that AHSCT is able to reduce the inflammatory milieu, to reset the immune balance and to promote the generation of a new auto-tolerant immune repertoire. However, some patients fail to remain in remission, and disease is re-activated after some time post-transplantation. This scenario indicates that further immunological interventions may be still required to improve AHSCT efficacy. Clear understanding of the operating immune mechanisms that contribute to specific clinical outcomes is vital to enable improvement of AHSCT protocols for autoimmune diseases.
