Stem Cell Therapy as a Treatment for Autoimmune Disease-Updates in Lupus, Scleroderma, and Multiple Sclerosis

Evidence for hematopoietic stem cell transplantation (HCT) in autoimmune disease has been building since the 1990s; however, many clinicians may not yet be aware of its applications to autoimmune disease. We review the basic tenets of HCT and evidence for autologous HCT in multiple sclerosis (MS), systemic sclerosis (SSc), and lupus with an emphasis on recent advanced phase trials.

In MS, the phase 3 randomized MIST trial and the phase 2 randomized ASTIMS trial demonstrated the efficacy of autologous HCT in refractory MS over disease-modifying therapies and mitoxantrone, respectively. In SSc, the phase 3 randomized ASTIS trial and the phase 2 randomized SCOT trial demonstrated the efficacy of autologous HCT in advanced SSc compared to cyclophosphamide. The evidence for HCT in autoimmune diseases continues to grow, particularly in MS and SSc. In lupus, large, comparative trials are still needed. Across autoimmune diseases, questions that still remain to be answered include optimizing patient selection to limit TRM, the appropriate use of MAC, and the necessity for graft manipulation. Furthermore, collaboration between disease-specific and transplant physicians is imperative to expand the appropriate use of HCT in routine clinical practice.