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Stem Cell Transplantation as Lupus Treatment

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February 1, 2006

About half of patients with severe lupus that does not respond to standard treatment and who undergo autologous stem cell transplantation to boost their immune system have substantial improvement of their disease after several years, according to preliminary research published in the February 1 issue of The Journal of the American Medical Association.

Autologous (using the body's own) stem cell transplantation includes eliminating defective lymphocytes (type of white blood cell involved in the immune system), often through chemotherapy, followed by infusion of blood cell-producing stem cells. 

The research was conducted by Richard K. Burt, M.D., and colleagues at the Feinberg School of Medicine. Burt is professor of medicine and chief of the division of immunotherapy for autoimmune diseases at the Feinberg School and at Northwestern Memorial Hospital.

Lupus is a multisystem autoimmune disease that, despite advances in immunosuppressive medical therapies, continues to cause significant illness and death among patients with active disease.

Burt and co-researchers wanted to determine whether autologous stem cell transplantation could be performed safely in patients with lupus that has been resistant to other therapies, and whether there is sufficient evidence of efficacy to justify a definitive randomized trial.

The Northwestern study included 50 patients who were enrolled from 1997 through early 2005.

The researchers found that of the 48 patients who underwent stem cell transplantation (two patients died before transplantation), with an average followup of about 2.5 years, the overall five-year survival was 84 percent. The probability of disease-free survival at five years was 50 percent. The longest continuous duration of remission has been 7.5 years. Treatment-related death was 2 percent (1 of 50). By intention to treat, treatment-related death was 4 percent (2 of 50).

“This trial provides the justification for a randomized study that compares autologous stem cell transplantation with continued standard of care. Through randomization, a cost-benefit analysis of autologous stem cell transplantation may be undertaken," Burt said.

Patients with treatment-resistant and active lupus involving multiple organ systems despite a relatively young age traditionally have a high disease-related mortality rate.

Continuing failing therapy for such patients is problematic but necessary to confirm that the increased acute risk of autologous stem cell transplantation would be offset by better disease control and improved long-term survival, especially because the standard of care for lupus is constantly changing with the introduction of newer therapeutic agents, Burt concluded.