Clinical Science
Autologous hematopoietic stem cell transplantation in refractory rheumatoid arthritis: Sustained response in two of four patients
Article first published online: 26 APR 2001
DOI: 10.1002/1529-0131(199911)42:11<2281::AID-ANR4>3.0.CO;2-E
Copyright © 1999 by the American College of Rheumatology
Additional Information
How to Cite
Burt, R. K., Georganas, C., Schroeder, J., Traynor, A., Stefka, J., Schuening, F., Graziano, F., Mineishi, S., Brush, M., Fishman, M., Welles, C., Rosen, S. and Pope, R. (1999), Autologous hematopoietic stem cell transplantation in refractory rheumatoid arthritis: Sustained response in two of four patients. Arthritis & Rheumatism, 42: 2281–2285. doi: 10.1002/1529-0131(199911)42:11<2281::AID-ANR4>3.0.CO;2-E
Publication History
- Issue published online: 26 APR 2001
- Article first published online: 26 APR 2001
- Manuscript Accepted: 17 APR 1999
- Manuscript Received: 19 FEB 1999
- Abstract
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- Cited By
Abstract
Objective
To investigate the safety and efficacy of immune ablation with subsequent autologous hematopoietic stem cell transplantation (HSCT) in severe rheumatoid arthritis (RA).
Methods
Four patients with refractory RA and poor prognostic indicators were treated. Stem cells were collected and lymphocytes were depleted by 2.3–4.0 logs. The conditioning regimen included cyclophosphamide (200 mg/kg), antithymocyte globulin (90 mg/kg), and, for 1 patient, total body irradiation (TBI) with 400 cGy. Improvement was evaluated according to the American College of Rheumatology (ACR) preliminary definition of improvement in RA (ACR 20), and also according to the ACR 50 and ACR 70 criteria.
Results
HSCT was well tolerated. Three patients fulfilled the ACR 70 criteria at 1 month and 3 months post-HSCT. One patient did not fulfill the ACR 20 criteria because of persistent joint tenderness, despite improvement of the joint swelling. At 6 months post-HSCT, 1 patient fulfilled the ACR 70 criteria and 1 fulfilled the ACR 50 criteria, and these 2 patients fulfilled the ACR 70 criteria at 9 months post-HSCT. The other 2 patients (including the patient who received TBI) did not meet the ACR 20 criteria at 6 months and 9 months post-HSCT. The only patient with followup of >9 months fulfilled the ACR 70 criteria at 20 months post-HSCT.
Conclusion
In this series, autologous HSCT was safe and effective in inducing major clinical response and maintained significant benefit for 2 patients at 9 months and 20 months posttreatment, respectively. Sustained response did not occur for 2 of 4 patients. A regimen dose-response effect may exist, but the addition of TBI did not prevent disease relapse for 1 of the patients. More aggressive T cell depletion of the autograft, use of a myeloablative regimen, or use of an allograft may be necessary to decrease relapse rates.

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