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Autologous hemopoietic stem-cell transplantation for children with refractory autoimmune disease

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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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Authors and Affiliations

  1. Department of Pediatric Immunology, Wilhelmina Children’s Hospital, University Medical Centre Utrecht, PO 85090, 3508AB, Utrecht, The Netherlands

    Nico M. Wulffraat MD, PhD, Lieke AM Sanders MD, PhD & Wietse Kuis MD, PhD

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  1. Nico M. Wulffraat MD, PhD
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  2. Lieke AM Sanders MD, PhD
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  3. Wietse Kuis MD, PhD
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Wulffraat, N.M., Sanders, L.A. & Kuis, W. Autologous hemopoietic stem-cell transplantation for children with refractory autoimmune disease. Curr Rheumatol Rep 2, 316–323 (2000). https://doi.org/10.1007/s11926-000-0069-8

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  • DOI: https://doi.org/10.1007/s11926-000-0069-8

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