Systemic lupus erythematosus (SLE) is an autoimmune disease in which the immune system attacks the body’s own tissues and organs, leading to damage to the joints, skin, kidneys, heart, lungs, blood vessels, and brain. There is no cure for SLE, but medications allow most people with lupus to lead healthy, active lives. However, some cases of lupus are very severe and do not respond well to these medications.

In a new study in the February 1, 2006 issue of the Journal of the American Medical Association , researchers found that stem cell transplantation along with high-dose chemotherapy benefited people with severe SLE that was not responding to standard treatment.

About the Study

Researchers from John Hopkins University identified 50 people with organ- or life-threatening SLE that was not responding to standard treatment. Before undergoing high-dose chemotherapy to repress white blood cells in the immune system, the participants had some of their own stem cells removed. After the chemotherapy was complete, the stem cells were transplanted back to make new immune cells replacing those that had been lost by the treatment. This same technique has been used for years in the treatment of certain life-threatening cancer with varying success.

The researchers followed the participants for up to 7.5 years to determine how the treatment affected the participants’ chances of survival and remission (a period of inactive disease). One participant died during the treatment phase, indicating a 2% (1 in 50) risk of treatment-related death. Half of the participants were in remission five years after the treatment, and 84% survived for at least five years. Overall, the participants’ scores on a test that measured their disease activity improved and remained significantly lower for up to five years after treatment.

This study was limited because it was relatively small and did not compare the treatment regimen with another treatment (or a placebo). It was designed to test the safety and effectiveness of stem cell transplantation, and determine whether a randomized trial comparing stem cell transplantation with standard treatment was justified.

How Does This Affect You?

This preliminary study suggests that the combination of high-dose chemotherapy and stem cell transplantation is a relatively safe treatment that may benefit people with severe lupus. But this treatment is not without its drawbacks. There is a risk of death and infection as a result of stem cell transplantation, and some patients who go into complete remission will have relapses later.

Whether stem cell transplantation will be used in practice for treating severe lupus remains to be seen. While the technique does not seem to be a cure for lupus, it may be a promising treatment for people who are not responding to other treatments. Future randomized trials will weigh the benefits and risks of this treatment, and help determine whether stem cell transplantation is superior to standard therapies in treating severe lupus.