Hematopoietic Cell Transplantation for Autoimmune Disease: Updates from Europe and the United States

Both genetic and environmental factors play a role in the development of autoimmune disease. Development of autoimmune disease in adulthood suggests that multiple immunizing events are required to break immune tolerance. It is proposed that autologous HCT, by “resetting” the immunological memory, may return the individual's immune system to a premorbid state, resulting in a prolonged clinical remission.

Improvements in serial Rodnan skin score (A), Health Assessment Questionnaire (B), Forced Vital Capacity, FVC (C) and Diffusion Capacity, DLCO (D) following immunoablation and autologous HCT. Gray solid lines depict individual patient parameters. Solid black lines are mean values and dashed lines represent the generalized estimating equation (GEE) for repeated measures. Mean Rodnan scores (0 is normal, 51 is maximal skin hardening throughout the body) decreased over time (P < .001). Improvements in mean Health Assessment Scores (0 is normal) were also significant (P < .001). The mean FVC values improved over time (P = .01), whereas the mean DLCO did not change significantly (P = .5). This research was originally published in Nash RA, McSweeney PA, Crofford LJ, et al. High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study. Blood, 2007;110:1386-1396. Copyright the American Society of Hematology.