Research shows promise for using stem cell transplantation to treat patients with severe lupus

January 31, 2006

About half of patients with severe lupus that was refractory to standard treatment and who underwent autologous stem cell transplantation to improve their immune system have substantial improvement in disease activity after several years, according to preliminary research published in the February 1 issue of JAMA.

Systemic lupus erythematosus (SLE) is a multisystem autoimmune disease that, despite advances in immunosuppressive medical therapies, continues to cause significant illness and death among patients with active disease, according to background information in the article. A more recent treatment is autologous hematopoietic stem cell transplantation (HSCT), in which stem cells from the patient are mobilized and re-infused into that patient to make new immune cells. HSCT includes eliminating defective lymphocytes (type of white blood cell involved in the immune system), often through chemotherapy, followed by infusion of hematopoietic stem cells (HSCs).

Richard K. Burt, M.D., of the Northwestern University Feinberg School of Medicine, Chicago, and colleagues conducted a study to determine whether HSCT could be performed safely in patients with SLE that has been resistant to other therapies, and whether there is sufficient evidence of efficacy to justify a definitive randomized trial. The study included 50 patients who were enrolled from April 1997 through January 2005.

The researchers found that of the 48 patients who underwent HSCT (2 patients died before transplantation), with an average followup of about 2.5 years, the overall 5-year survival was 84 percent. The probability of disease-free survival at 5 years was 50 percent. The longest continuous duration of remission has been 7.5 years. Treatment-related death was 2 percent (1/50). By intention to treat, treatment-related death was 4 percent (2/50).

"This trial provides the justification for a randomized study that compares autologous HSCT with continued standard of care. Through randomization, a cost-benefit analysis of HSCT may be undertaken. 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 HSCT 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," the authors conclude.
(JAMA. 2006;295:527-535. Available pre-embargo to the media at Editor's Note: The authors wish to thank the BraveWings Foundation and Ginger's Tomorrow Foundation for financial and patient support.

Editorial: High-dose Cyclophosphamide and Stem Cell Transplantation for Refractory Systemic Lupus Erythematosus

In an accompanying editorial, Michelle Petri, M.D., M.P.H., and Robert Brodsky, M.D., of the Johns Hopkins University School of Medicine, Baltimore, comment on the study on treating lupus with stem cell transplantation.

"The original hope of stem cell transplantation was 'going for the cure' in patients with severe lupus. The remission rates reported with use of high-dose cyclophosphamide with stem cell transplantation, as reported by Burt et al, or without autologous stem cells, do not necessarily represent 'cure'. Many patients continue to have lupus autoantibodies and with long-term follow-up, some patients with initial complete remissions will have late relapses. However, in these studies therapy was given as a 'salvage regimen' to patients with lupus who had failed or were [resistant to treatment] to multiple other therapies. Thus, as in the report by Burt et al the therapy offered substantial benefit-with either partial or complete response-to the majority of patients," they write. "Whether this approach represents a definitive advance over more conventional immunosuppressive therapies will need to be answered in randomized controlled trials." (JAMA. 2006;295:559-560. Available pre-embargo to the media at

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