Stem cell transplantation for autoimmune disorders. Refractory juvenile idiopathic arthritis.
Since 1997, haematopoietic stem cell transplantation (HSCT) has been applied as an experimental procedure in more than 50 children with refractory juvenile idiopathic arthritis. We describe here follow-up data on 34 children with juvenile idiopathic arthritis, treated with HSCT in order to evaluate its feasibility, safety and efficacy. Data were collected on immunological reconstitution, complications and key rheumatological parameters. The clinical follow-up of the children ranged from 12 to 48 months. Eighteen of the 34 patients achieved a drug-free complete remission. Seven of these patients had previously failed treatment with anti-tumour necrosis factor-alpha. Six of the 34 patients showed a partial response (ranging from 30 to 70%), and 7 of the 34 patients showed a complete relapse of disease. Infectious complications were frequently seen. There were three cases of transplant-related mortality and two cases of disease-related mortality. It is still unclear why especially patients with systemic juvenile idiopathic arthritis are at risk of episodes of reactive haemophagocytosis.
Wulffraat
Nico M
NM
Department of Paediatric Immunology and Rheumatology, Wilhelmina Children's Hospital, University Medical Center Utrecht, P.O. Box 85090, Utrecht 3508 AB, The Netherlands. n.wulffraat@wkz.azu.nl
de Kleer
Ismee M
IM
Prakken
Berent J
BJ
Kuis
Wietse
W
D001171:Arthritis, Juvenile / Q000628:therapy*; D002648:Child; D005500:Follow-Up Studies; D006801:Humans; D018579:Patient Selection; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D013997:Time Factors; D014182:Transplantation, Autologous