A Novel but Potentially Risky Therapy for Severe Rheumatoid Arthritis

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The summary below is from the full report titled "High-Dose Chemotherapy and Syngeneic Hemopoietic Stem-Cell Transplantation for Severe, Seronegative Rheumatoid Arthritis." It is in the 5 October 1999 issue of Annals of Internal Medicine (volume 131, pages 507-509). The authors are G. McColl, H. Kohsaka, J. Szer, and I. Wicks.

What is the problem and what is known about it so far?

Rheumatoid arthritis is a form of autoimmune arthritis. Autoimmune means that the disease results when a person develops immune reactions against substances in his or her own body. Rheumatoid arthritis can be disabling and difficult to treat. Some researchers think it might make sense to treat autoimmune diseases with strong drugs to wipe out the abnormal immune cells (chemotherapy), followed by replacement of the abnormal blood cells with early forms of normal ones (stem-cell transplantation). The normal stem cells in this study come from a person who does not have autoimmune disease, but could also come from the patient with rheumatoid arthritis. This kind of treatment is completely different from the usual kinds of rheumatoid arthritis treatment, but is widely used in cancer treatment.

Why did the researchers do this particular study?

The researchers describe what happened when they treated one patient with severe rheumatoid arthritis with chemotherapy and stem cell transplantation, using an identical disease-free twin as the stem cell donor.

Who was studied?

A 39-year-old man with severe rheumatoid arthritis that had damaged his wrist, ankle, and shoulder joints. He did not get better with any of the many treatments he had received.

How was the study done?

The patient received chemotherapy with two drugs, cyclophosphamide and anti-thymocyte globulin, which destroyed the immune cells. His doctors then took stem cells from his identical twin brother (who did not have rheumatoid arthritis) using a procedure called leukapheresis and gave them to the man with rheumatoid arthritis. To perform leukapheresis, the doctors use an intravenous line to connect the person who is donating the stem cells to a machine that filters the cells from the blood.

What did the researchers find?

For two years after the procedure, the patient had recovered enough that he could swim, could ride a bicycle, and had no symptoms from rheumatoid arthritis. He was also not receiving any other treatment for his arthritis. His twin brother was also well.

What were the limitations of the study?

This report describes a single case, so it is uncertain whether other persons with rheumatoid arthritis would respond as well to this type of treatment. This type of therapy is highly experimental, expensive, and quite risky. Bone marrow makes the cells that help the body fight infections, so potentially deadly infections may occur before the bone marrow cells begin to grow back and long term side effects may occur.

What are the implications of the study?

This study encourages further research to see whether similar favorable results are possible in other patients with rheumatoid arthritis.