REVIEW - Stem cell transplantation in RA

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Autoimmunity. 2008 Oct 28:1.

Stem cell transplantation in rheumatoid arthritis.

Snowden JA, Kapoor S, AG.

Department of Haematology, Sheffield Teaching Hospitals NHS Trust,

Sheffield, UK.

The therapeutic potential of high dose cytotoxic therapy and stem cell

transplantation (SCT) in severe rheumatoid arthritis (RA) was

originally supported by animal studies and serendipitous clinical

cases where allogeneic and autologous procedures were shown to

ameliorate and potentially cure the disease. Phase I and Phase II

clinical studies established the feasibility, safety and efficacy of

autologous stem cell mobilisation and transplantation. Although it was

clear that the effects of high dose chemotherapy and autologous SCT

could safely achieve profound responses, sustained control of disease

usually required the reintroduction of disease modifying agents.

Responses were improved with dose escalation of the conditioning

regimen, and also with post-SCT therapy, such as rituximab, but were

not observed with graft manipulation. Phase III studies were

attempted, but recruitment was compromised by the increasingly

widespread use of biological anti-rheumatic agents. Autologous SCT is

now only reasonably considered in relatively rare patients whose

disease has resisted conventional and biological treatments, and small

numbers of cases continue to be registered with the EMBT. Occasional

patients treated with allogeneic and syngeneic SCT continue to

stimulate academic interest, particularly as some appear to be cured,

but significant logistical and toxicity issues mean that routine and

widespread application is unrealistic. In summary, SCT continues to

have a limited therapeutic potential in rare patients with RA

refractory to modern therapy and sufficient fitness for the procedure.

From a scientific perspective, ablation of the dysfunctional

rheumatoid immune system and its reconstruction with SCT has provided

useful insights into the pathophysiology of RA.

PMID: 18958746

http://www.ncbi.nlm.nih.gov/pubmed/18958746

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