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Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement

As seen from both patients’ and physicians’ points of view, there is wide agreement that systemic sclerosis (SSc) is one of the autoimmune disorders with the highest morbidity and mortality rates. In this review, we will follow the evolution of a new approach to its treatment. Support for using hematopoietic stem cell transplantation (HSCT) for SSc arose from seminal studies of genetic and antigen‐induced experimental models of autoimmune disease that demonstrated that high‐dose immunosuppression followed by either allogeneic (same species) or autologous (self) bone marrow transplantation (BMT) could prevent and even reverse damage from autoimmune diseases. Three decades after these initial preclinical observations, our understanding of the therapeutic potential of immune restoration following autologous HSCT has deepened, and the clinical evidence for its application in scleroderma has broadened.

In this review we will examine the outcome of conventional therapy for scleroderma lung disease; detail the techniques, toxicities, and results of HSCT for SSc; and explore the biology of immune restoration following autologous transplantation. We will compare the design and outcomes of randomized trials comparing HSCT with cyclophosphamide (CYC) treatment and formulate criteria for the timely referral of patients with scleroderma lung disease for HSCT.