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Editorial |

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

Dinesh Khanna, MD, MS1; George E. Georges, MD2; Daniel R. Couriel, MD, MS3
[+] 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.
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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

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