Autologous hematopoietic stem cell transplantation for 3 patients with severe juvenile rheumatoid arthritis
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Published on: October 16, 2016
Tomoko Kishimoto1, Takashi Hamazaki, Masahiro Yasui
1Department of Pediatrics, Osaka Medical Center and Research Institute for Maternal and Child Health, Izumi City, Osaka, Japan.
Insights
Autologous CD34+ stem cell transplantation shows promise for treating refractory juvenile rheumatoid arthritis (JRA). Two of three patients achieved remission, indicating a safe and feasible treatment option for JRA.
Area of Science:
- Immunology
- Hematology
- Pediatric Rheumatology
Background:
- Juvenile rheumatoid arthritis (JRA) is a chronic autoimmune disease affecting children.
- Refractory JRA cases often have limited treatment options.
- Autologous stem cell transplantation is an emerging therapy for autoimmune diseases.
Observation:
- Three patients with systemic JRA refractory to conventional therapies underwent autologous CD34+ stem cell transplantation.
- Case 1 received bone marrow-derived CD34+ cells with cyclophosphamide and antithymocyte globulin (ATG) preconditioning, experiencing a disease flare.
- Cases 2 and 3 received peripheral blood-derived CD34+ cells with etoposide (VP16), thiotepa, and ATG preconditioning, achieving complete remission.
Findings:
- Autologous CD34+ stem cell transplantation demonstrated feasibility in refractory JRA.
- A modified preconditioning regimen (VP16, thiotepa, ATG) in cases 2 and 3 led to complete remission.
- The treatment appears safe, improving quality of life for patients with refractory JRA.
Implications:
- Autologous CD34+ stem cell transplantation is a potential therapeutic strategy for refractory JRA.
- Further research with larger cohorts and longer follow-up is necessary to confirm efficacy and long-term outcomes.
- This approach may offer a new avenue for managing severe, treatment-resistant JRA.
Abstract:
We performed autologous CD34+ stem cell transplantation in 3 patients with juvenile rheumatoid arthritis (JRA) refractory to conventional treatment. All patients had systemic type JRA. In case 1 (a 3-year-old boy), purified CD34+ cells from bone marrow were transplanted after a preconditioning regimen consisting of cyclophosphamide (200 mg/kg) and antithymocyte globulin (ATG) (40 mg/kg). However, the disease flared soon after transplantation. In case 2 (a 13-year-old girl) and case 3 (a 21-year-old woman), a preconditioning regimen consisting of etoposide (VP16) (2 g/m2), thiotepa (300 mg/m2), and ATG (40 mg/kg) was followed by transplantation of purified CD34+ stem cells harvested from peripheral blood mononuclear cells. The patients in cases 2 and 3 attained complete remission without any medication. Thus for patients with refractory JRA, autologous CD34+ cell transplantation appears to be a safe and feasible choice of treatment in terms of good quality of life. However, a greater number of patients and a longer observation period are needed before definitive conclusions can be drawn.
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