Lupus, an autoimmune disease in which immune cells attack an individual's own organs, affects an estimated 1.5 million people, mostly young females. "For this study, we enrolled patients who had either life- or organ-threatening lupus and had exhausted all available treatment options," says lead author Richard Burt, MD, chief, Division of Immunotherapy for Autoimmune Diseases, Northwestern Memorial Hospital in Chicago and associate professor of Medicine at Northwestern University's Feinberg School of Medicine. "We found that within an experienced center, high-dose chemotherapy and autologous stem cell transplant may be performed safely and result in disease remission and improvement or salvage of residual organ function in the majority of patients."
The study, which was launched in 1997 when Dr. Burt performed the country's first stem cell transplant to treat lupus, enrolled 50 patients from 20 states and ran through January 2005. The authors conclude that that the findings provide the justification to launch a randomized study that would compare autologous stem cell transplant with continued standard of care.
The stem cell transplant process used in the study is similar to that done to treat some forms of cancer. The patients' own bone marrow stem cells were harvested from their blood. These cells, which can become different kinds of blood and immune system cells in the body, were then separated from the other blood cells. Next, in a process that usually requires a few weeks of hospitalization, patients immune systems were virtually destroyed through high doses of chemotherapy. Then the cleansed stem cells were returned to the bone marrow to repopulate the marrow and body in an effort to regenerate a healthier immune system. "The idea is that if you turn back the clock and let the immune system heal itself, the patient should have a chance of ending up without the disease," says Dr. Burt.
A European multi-center trial published in 2004 reported a similar success rate; however the treatment-related mortality rate was much higher at 13 percent, compared to a 2 percent rate in this study. The authors note that the toxicity of the treatment is a result of patient selection, the conditioning regimen and supportive care during and after transplantation. "It is doubtful our lower treatment mortality rate was due to patient selection, as our patients were very ill," says co-author Yu Oyama, MD, an autoimmune disease specialist at Northwestern Memorial Hospital and assistant professor, Division of Immunotherapy for Autoimmune Diseases at the Feinberg School of Medicine.
"A center effect on survival has also been reported for stem cell transplantation in malignancies," says Dr. Burt. "However, it's important to note that the patients in this study are not comparable to those with malignancies. The patients in this study had organ dysfunction and were immunocompromised for a long time, conditions which often rule out stem cell transplant for cancer patients because it would be too dangerous. This highlights the importance for a medical center to have experience when treating lupus patients with high-dose chemotherapy and stem cell transplant."
Lupus is a chronic, inflammatory, autoimmune disease that mainly affects women of child-bearing age. Its symptoms range from unexplained fever, swollen joints and skin rashes to severe damage of the kidneys, lungs or central nervous system. Lupus is three times more common -- and is frequently more severe -- in African Americans and Hispanics/Latinos. Studies show these groups also experience more complications of lupus, including kidney failure for both and neurological problems for African Americans.
"Fortunately, the majority of patients with lupus can be successfully managed with our available medical therapies," says co-author Walter G. Barr, MD, a rheumatologist at Northwestern Memorial Hospital and professor of Medicine in the Division of Rheumatology at Northwestern University's Feinberg School of Medicine. "However, for the very severely ill subset of lupus patients who have failed conventional therapies, stem cell transplantation provides a promising new alternative."
This study received funding support from the BraveWings Foundation and Ginger's Tomorrow Foundation.
About Northwestern Memorial Hospital
Northwestern Memorial Hospital is one of the country's premier academic medical centers and is the primary teaching hospital of Northwestern University's Feinberg School of Medicine. Northwestern Memorial and its Prentice Women's Hospital and Stone Institute of Psychiatry have 744 beds and more than 1,400 affiliated physicians and 5,000 employees. Providing state-of-the-art care, Northwestern Memorial is recognized for its outstanding clinical and surgical advancements in such areas as cardiothoracic and vascular care, gastroenterology, neurology and neurosurgery, oncology, organ and bone marrow transplantation and women's health.
Northwestern Memorial was the sole recipient of the prestigious 2005 National Quality Health Care Award and eight of its medical specialties are listed in this year's U.S. News & World Report's issue of "America's Best Hospitals." The hospital also has been cited as one of the "100 Best Companies for Working Mothers" by Working Mother magazine six consecutive years and has been chosen by Chicago-area consumers for more than a decade as their "most preferred hospital" in National Research Corporation's annual survey.
Journal
JAMA