Related Experiment Video
Updated: Jun 26, 2026
In Vivo Osteo-organoid Approach for Harvesting Therapeutic Hematopoietic Stem/Progenitor Cells
Published on: February 16, 2024
Current perspectives of autologous stem cell transplantation for severe Juvenile Idiopathic Arthritis
N M Wulffraat1, E M van Rooijen, R Tewarie
1Department of Pediatric Immunology, University Medical Center Utrecht, Wilhelmina Children's Hospital, Utrecht, The Netherlands. n.wulffraat@umcutrecht.nl
Insights
Autologous stem cell transplantation (ASCT) offers a favorable long-term outcome for children with drug-resistant Juvenile Idiopathic Arthritis (JIA). While late relapses can occur, they are often manageable, and newer regimens show promising remission rates.
Area of Science:
- Pediatric Rheumatology
- Hematology
- Immunology
Background:
- Juvenile Idiopathic Arthritis (JIA) affects children, with many responding to current treatments like antirheumatic drugs, corticosteroids, and biologics targeting TNF, IL-1, or IL-6.
- A subset of JIA patients exhibit resistance to conventional therapies, experiencing severe, debilitating, and potentially life-threatening disease.
- Autologous stem cell transplantation (ASCT) has been utilized since 1997 for these refractory JIA cases.
Purpose of the Study:
- To report the long-term outcomes of an initial cohort of children with treatment-resistant JIA who underwent ASCT.
- To evaluate the efficacy and safety of different ASCT conditioning regimens in pediatric patients with severe JIA.
Main Methods:
- Analysis of long-term outcomes in children with resistant JIA treated with ASCT.
- Initial cohort received conditioning with Cyclophosphamide, anti-thymocyte globulins, and low-dose Total Body Irradiation (TBI).
- More recent ASCT protocols utilized a fludarabine-based regimen instead of low-dose TBI.
- Review of outcomes for two cases of allogeneic transplant in JIA patients.
Main Results:
- The initial ASCT cohort (Cyclophosphamide, anti-thymocyte globulins, low-dose TBI) showed favorable responses, with a 50-55% drug-free remission rate.
- Late relapses were observed in some patients years after ASCT, though these were generally less severe and treatable with conventional drugs.
- Four recent JIA patients treated with a fludarabine-containing ASCT regimen achieved drug-free remission at 4-5 year follow-up.
- Two JIA patients undergoing allogeneic transplant showed disease remission and medication tapering at 1 and 3-year follow-ups.
Conclusions:
- ASCT is a valuable treatment option for children with drug-resistant Juvenile Idiopathic Arthritis.
- Favorable long-term outcomes support the continued use of ASCT for severe, refractory JIA.
- The fludarabine-based conditioning regimen shows promising results for achieving sustained remission in JIA patients.
Abstract:
The majority of children with Juvenile Idiopathic Arthritis can nowadays be treated adequately. However despite the use of combinations of antirheumatic drugs, corticosteroids and the newer so called biologicals (blocking the TNF, Interleukin 1 or Interleukin 6 pathways) a proportion of children with arthritis remain resistant also to these therapies and suffer from a very severe, debilitating and potentially fatal disease. For such children autologous stem cell transplantation (ASCT) is successfully performed since 1997. Here we describe the long term outcome of the initial cohort of children with resistant Juvenile Idiopathic arthritis, treated with ASCT. The initial cohort of children was treated with a conditioning regimen containing Cyclophosphamide, anti thymocyte globulins and low dose Total Body irradiation. Overall favourable responses were seen, with a drug free remission rate of 50-55 %. In the more recent years late relapses were noted with lower percentages for drug free long term outcome. Special emphasis is given on 2 cases showing very late relapses, occurring after 7 and 9 years. The observed relapses are often less severe compared to the situation before SCT and can be treated successfully with conventional drugs in the majority of cases. More recently, ASCT was performed in 4 JIA children with a fludarabin containing regimen in stead of low dose TBI. With a 4 to 5 year follow up, these 4 patients are all in drug free full remission. Allogeneic transplant with an HLA matched family donor was reported in 2 JIA cases. Follow up of 1 and 3 year is sofar show clinical disease remission and tapering of medition. In conclusion, given the favourable long term outcome, SCT remains a valuable treatment option for children with drug resistant JIA.
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...
01:21Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
01:22iPS Cell Differentiation
01:21Satellite Stem Cells and Muscular Dystrophy
01:19Mesenchymal Stem Cells
01:17Stem Cell Culture