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
Khanna D, Georges GE, Couriel DR. Autologous Hematopoietic Stem Cell Therapy in Severe Systemic Sclerosis: Ready for Clinical Practice? JAMA. 2014;311(24):2485–2487. doi:10.1001/jama.2014.6369
Coronavirus Resource Center
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: