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Göteborgs universitets publikationer

Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity

Författare och institution:
I. M. de Kleer (-); D. M. Brinkman (-); A. Ferster (-); M. Abinun (-); P. Quartier (-); J. Van Der Net (-); R. ten Cate (-); L. R. Wedderburn (-); G. Horneff (-); J. Oppermann (-); F. Zintl (-); H. E. Foster (-); A. M. Prieur (-); Anders Fasth (Institutionen för kvinnors och barns hälsa, Avdelningen för pediatrik); M. A. van Rossum (-); W. Kuis (-); N. M. Wulffraat (-)
Publicerad i:
Ann Rheum Dis, 63 ( 10 ) s. 1318-26
0003-4967 (Print)
Artikel, refereegranskad vetenskaplig
Sammanfattning (abstract):
OBJECTIVE: To evaluate the safety and efficacy of autologous stem cell transplantation (ASCT) for refractory juvenile idiopathic arthritis (JIA). DESIGN: Retrospective analysis of follow up data on 34 children with JIA who were treated with ASCT in nine different European transplant centres. Rheumatological evaluation employed a modified set of core criteria. Immunological reconstitution and infectious complications were monitored at three month intervals after transplantation. RESULTS: Clinical follow up ranged from 12 to 60 months. Eighteen of the 34 patients (53%) with a follow up of 12 to 60 months achieved complete drug-free remission. Seven of these patients had previously failed treatment with anti-TNF. Six of the 34 patients (18%) showed a partial response (ranging from 30% to 70% improvement) and seven (21%) were resistant to ASCT. Infectious complications were common. There were three cases of transplant related mortality (9%) and two of disease related mortality (6%). CONCLUSIONS: ASCT in severely ill patients with JIA induces a drug-free remission of the disease and a profound increase in general wellbeing in a substantial proportion of patients, but the procedure carries a significant mortality risk. The following adjustments are proposed for future protocols: (1) elimination of total body irradiation from the conditioning regimen; (2) prophylactic administration of antiviral drugs and intravenous immunoglobulins until there is a normal CD4+ T cell count.
Ämne (baseras på Högskoleverkets indelning av forskningsämnen):
Arthritis, Juvenile Rheumatoid/immunology/*therapy, Child, Child, Preschool, Female, Follow-Up Studies, Graft Survival, Health Status Indicators, Humans, Infant, Male, Opportunistic Infections/etiology, Patient Selection, Retrospective Studies, Severity of Illness Index, Stem Cell Transplantation/adverse effects/*methods, Survival Analysis, T-Lymphocyte Subsets/immunology, Transplantation Conditioning/methods, Transplantation, Autologous, Treatment Outcome
Postens nummer:
Posten skapad:
2007-10-24 13:35

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