Hematopoietic Stem Cell Transplant for Rheumatoid Arthritis (RA)

June 24, 2002

Autoimmune diseases are defined as diseases in which the bodies immune

system has difficulty recognizing the rest of the body as " self " and

therefore, thinking it is the enemy, it can mount an immune response, or

attack, much as it would against outside agents such as viruses or bacteria.

Stem cell transplantation has been used in cancer with enough success that

enthusiasm has been created in other fields, such as rheumatology.

The ability to transfer autoimmune disease to normal animals by the infusion

of specialized cells from the bone marrow (hematopoietic stem cells, or HSC)

opens new avenues of research to investigate the possible cause and

mechanisms of autoimmunity. Understanding these mechanisms could lead to the

development of treatment applications in patients with such diseases.

Improvement in the understanding of rheumatoid arthritis has suggested the

need for more intensive therapy in order to decrease disability, decrease

mortality and improve quality of life. Some patients require a combination

of multiple medications, and HSC transplant could achieve such dream-like

goals.

How does HSCT (HSC Transplant or Reconstitution) work?

Destroying cells that cause autoimmunity and replacing them with a normal

immune system or at least reducing the level to a point of creating enough

self-tolerance (lack of allergy or immunity to self) to avoid disease.

Another mechanism is altering circuits within the immune system that have

gone wrong and thus created a problem with self-recognition.

Who qualifies for HSCT?

Because of the complexity of this form of therapy, patient selection is

extremely important, and at the present time should only be offered to those

who have failed all conventional forms of therapy or those whose disease

course can be predicted as one that will cause significant premature

mortality.

One significant issue is how to identify patients at risk for early death or

disability during the early stages of disease.

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