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Paediatric rheumatologists in two specialist centres in the UK are calling for international collaboration to refine a promising treatment for children with severe juvenile idiopathic arthritis (JIA) when other options have failed. The treatment is intense immunosuppression followed by autologous haemopoietic stem cell transplantation (ASCT), and future steps are understanding how it works in molecular terms and predicting who will benefit so that treatment can start before severe complications set in.
More than 45 children worldwide have had ASCT, and the signs are hopeful. In continental Europe results for 45 children, which are being written up, show about half in “complete” (drug free) remission, and in a published series 16 of 29 children were in drug free remission three years after treatment and eight were in partial remission or relapse, needing drug treatment.
ASCT carries with it appreciable risks of death and fatal complications. So selecting patients is crucial and is subject to consensus guidelines. Eligible children are those who show JIA, failed drug treatment, and drug toxicity; their disease must be controlled; and they must have no fevers or infections. Additionally, the family and the children are counselled carefully about risks and benefits against other treatments and about the lengthy, stressful, nature of the treatment and the uncertainty of the outcome. The psychological demands on patients and families are appreciable.
Two children in the UK have been treated so far, with good results. In true collaborative spirit the data will be added to the European Blood and Marrow Transplantation (EBMT) database.
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