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

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.

• Authors: I M De Kleer, D M C 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, A Fasth, M A J Van Rossum, W Kuis, N M Wulffraat
• Journal: Annals of the rheumatic diseases (Ann Rheum Dis) Vol. 63 Issue 10 Pg. 1318-26 (Oct 2004) ISSN: 0003-4967 [Print] England
• PMID: 15361393 (Publication Type: Evaluation Study, Journal Article, Research Support, Non-U.S. Gov't)

Topics

• Arthritis, Juvenile (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