Autologous hemopoietic stem-cell transplantation for children with refractory autoimmune disease |
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Authors: | Nico M Wulffraat MD PhD Lieke AM Sanders MD PhD Wietse Kuis MD PhD |
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Institution: | (1) Department of Pediatric Immunology, Wilhelmina Children’s Hospital, University Medical Centre Utrecht, PO 85090, 3508AB Utrecht, The Netherlands |
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Abstract: | Autologous stem cell transplantation (ASCT) has been proposed as a possible treatment for severe autoimmune diseases such
as rheumatoid arthritis (RA), multiple sclerosis (MS), systemic sclerosis, and systemic lupus erythematosus (SLE). To date,
more than 250 patients with various autoimmune disorders have undergone an ASCT since 1996. Among them, there is a very limited
number of children. This review summarizes the experience with ASCT for pediatric rheumatic diseases. Most reported cases
concern juvenile idiopathic arthritis (JIA). Experience with ASCT for childhood SLE, Scleroderma, or Dermatomyositis is very
limited. To date, 12 children with severe systemic or polyarticular JIA, all with progressive disease activity despite the
use of corticosteroids, MTX, CsA, or Cyclophosphamide were treated in our center with ASCT. Rheumatologic follow-up at 3-month
intervals up to 36 months showed a marked decrease in arthritis severity as expressed by the core-set criteria for juvenile
chronic arthritis (JCA) activity. However, these children remain at risk for severe viral infections due to the prolonged
lymfopenia. ASCT in this severely ill patient group induces a very significant and drug-free remission of the disease, but
carries a significantly risk of developing fatal MAS. |
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