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Autologous haematopoietic stem cell transplantation in juvenile idiopathic arthritis
  1. L R Wedderburn1,
  2. M Abinun2,
  3. P Palmer3,
  4. H E Foster4
  1. 1Rheumatology Unit, Institute of Child Health, UCL and Great Ormond Street Hospital NHS Trust, London, UK
  2. 2Department of Paediatric Immunology, Newcastle General Hospital, Newcastle Hospitals NHS Trust, Newcastle upon Tyne, UK
  3. 3Children’s Bone Marrow Transplantation Unit, Newcastle General Hospital, Newcastle Hospitals NHS Trust, Newcastle upon Tyne, UK
  4. 4Departments of Rheumatology and Child Health, University of Newcastle upon Tyne and Newcastle Hospitals NHS Trust, Newcastle upon Tyne, UK
  1. Correspondence to:
    Dr L R Wedderburn, Rheumatology Unit, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK;

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A powerful strategy when other treatments have failed

Despite modern therapies, juvenile idiopathic arthritis (JIA) remains a significant cause of morbidity in children. The long term outcome of JIA is variable. Thus, while the prognosis for many children with JIA is good, over one third of children have ongoing active disease into adulthood with sequelae from chronic inflammation.1,2 Patients with systemic or polyarticular onset JIA tend to have a worse prognosis, even with the early use of disease modifying antirheumatic drugs (DMARDS). These patients have considerable morbidity from joint damage, osteoporosis, growth retardation, psychosocial morbidity, reduced quality of life, and educational or employment disadvantage.2,3

The concept of treating severe autoimmune disease by using intense, high dose immunosuppression to remove autoreactive lymphocytes, followed by rescue with haematopoietic stem cells, is based on the tenet that the immune system plays a pivotal role in such diseases. Support for this concept came from animal models, in which both spontaneous and induced autoimmune pathologies can be treated by lymphoablative/myeloablative treatment followed by stem cell rescue.4 Early observations that chemotherapy treatment of malignancy in patients with concurrent autoimmunity could induce remission of autoimmune symptoms, led physicians to attempt haematopoietic transplantation for such disorders, initially in rheumatoid arthritis (RA), systemic lupus erythematosus (SLE,) and multiple sclerosis (MS).5

In less than five years data have been collected on almost 400 patients who have undergone stem cell transplantation for autoimmune disorders.6 The majority have received autologous rather than allogeneic stem cells, since autologous transplantation carries lower mortality, and avoids the problems of graft matching and graft versus host disease (GVHD). Discussion continues about the potential curative nature of the use of allogeneic therapy,7,8 although some data suggest that arthritis can relapse even after allogeneic transplantation.9 This review concentrates …

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