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Stem cell transplant for refractory JIA

Clinical bottom line

Replacing T lymphocytes with an autologous stem cell transplant has hazards. In this report of initial treatments, some children died, and others had serious infections. But rheumatological results were good in many children.


In a small proportion of children with juvenile idiopathic arthritis, the disease does not respond to conventional therapy with methotrexate or corticosteroids, and even anti-TNF agents do not always help. The result is severe joint destruction, growth retardation, and adverse events of long-term use of immunosuppressives, combined with psychological problems.

Another new treatment is the use of intense immunosuppression with regeneration of naive T lymphocytes following autologous haemopoietic stem cell transplantation (ASCT). ASCT has been used on over 40 children, and here we have results from 34 in a retrospective cohort analysis.


IM de Kleer et al. Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity. Annals of the Rheumatic Diseases 2004 63: 1318-1326.


Thirty-four children received a transplant in nine different European centres, and who were entered in a specialised register of such patients, with sufficient detail. Before transplantation all children had polyarticular disease with erosions, osteoporosis, and stunted growth. All centres had used agreed inclusion and exclusion criteria for patients to have ASCT, and these included failure to respond to high-dose parenteral methotrexate, plus one other disease modifying agent, or unacceptable toxicity from them. Failure to respond to anti-TNF treatment was a later, additional, criterion.


Complete remission occurred in 18/34 (53%) children with a follow up of 12 to 60 months. No response was noted in seven children (21%); two of these children died 13 and 16 months after the transplant. Six children showed a partial response(18%)

After transplantation, most (70%) of children developed at least one infection. Six children had septicaemia. Three children died shortly after the transplant of infectious complications, and the total mortality was 5/34 children.

Over four years following transplantation, there were improvements in wellbeing, disability, active joint count, ESR, and pain. All seemed to be in the direction of more benefit over time.


Detailed investigation and reporting of small numbers of patients with a relatively rare but dangerous condition, faced with a hopeful but somewhat hazardous therapy is just what is needed. This study and report fulfils the requirements well.

There were and are obvious dangers, and not all children responded well. However, the report tells us that the eight children who entered after fulfilling the additional criterion of failing anti-TNF therapy did particularly well, and perhaps this bodes well for the future.