The trial, conducted between February 1997 and January 2005, was discussed by Richard K. Burt, MD, Associate Professor and Chief, Division of Immunotherapy and Autoimmune Diseases, Northwestern University Feinberg School of Medicine, and Northwestern Memorial Hospital, Chicago, Illinois, United States.

The researchers enrolled 50 patients who had SLE that was refractory to standard immune suppressive therapies and had life-threatening disease with involvement of vital organs or tissue.

The investigators mobilized peripheral blood stem cells with cyclophosphamide and granulocyte colony-stimulating factor (G-CSF) and enriched the graft by ex vivo immuno-selection for CD34. Patients received a non-myeloablative conditioning regimen consisting of intravenous cyclophosphamide and equine anti-thymocyte globulin.

After a mean follow-up of 29 months, none of the 48 patients who underwent transplantation showed treatment related mortality. The overall survival rate was 84%.

The calculated probability of disease-free survival at 5 years post-transplant was 50%.

Dr. Burt reported significant improvements in several markers of disease activity, including anti-nuclear antigen, anti ds-DNA and pulmonary function. He noted that none of six patients who were dependent on oxygen at enrollment required oxygen post-transplantation.

The rationale for the treatment strategy was to reset the immune system in a safe manner, using a non-myeloablative approach, Dr. Burt explained.

"An important part of our research is to fine-tune our methods for performing SCT in patients with autoimmune disease, which may differ significantly from methods used in the treatment of cancers such as leukemia and lymphoma," he said.

Transplantation of autologous stem cells results in a shorter period of neutropenia, further increasing the safety of the treatment for the patient, Dr. Burt said.

In other presentations, Northwestern University researchers also reported preliminary results of autologous hematopoietic SCT for several other auto-immune disorders, including multiple sclerosis, scleroderma and Crohn's disease. Larger controlled randomized trials for these conditions are planned.

The conference was sponsored by Roche.


[Presentation title: Non-Myeloablative Autologous Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus. Abstract 19]

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