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June 25, 2014

Autologous Hematopoietic Stem Cell Therapy in Severe Systemic Sclerosis: Ready for Clinical Practice?

Author Affiliations
  • 1Division of Rheumatology, Department of Internal Medicine, University of Michigan, Ann Arbor
  • 2Fred Hutchinson Cancer Research Center, University of Washington, Seattle
  • 3Blood and Marrow Transplantation Program, Department of Internal Medicine, University of Michigan, Ann Arbor
JAMA. 2014;311(24):2485-2487. doi:10.1001/jama.2014.6369

Systemic sclerosis is a life-threatening disease that includes a subset of patients with diffuse cutaneous systemic sclerosis (defined as widespread skin involvement) and internal organ involvement and has a mortality of 30% to 50% at 5 years.1 Conventional-dose immunosuppressive therapy has limited efficacy in preventing disease progression, and there are few data to support survival benefit.2 High-dose immunosuppression is an alternative to conventional-dose immunosuppression. Despite the increased risk of toxicity, the rationale for high-dose immunosuppression is that systemic sclerosis is an autoimmune disorder that progressively damages internal organs and is associated with chronic inflammation and systemic immunological abnormalities.3 Studies of high-dose immunosuppression (with autologous hematopoietic stem cell transplantation [HSCT]) in patients with systemic sclerosis have demonstrated efficacy in preventing disease progression,1,4 and a single-center trial (Autologous Non-myeloablative HSCT Compared With Pulse Cyclophosphamide Once per Month for Systemic Sclerosis [ASSIST]) demonstrated superiority of autologous HSCT over conventional therapy.4