Allogeneic marrow transplantation in patients with severe systemic sclerosis: Resolution of dermal fibrosis
Version of Record online: 26 MAY 2006
Copyright © 2006 by the American College of Rheumatology
Arthritis & Rheumatism
Volume 54, Issue 6, pages 1982–1986, June 2006
How to Cite
Nash, R. A., McSweeney, P. A., Nelson, J. L., Wener, M., Georges, G. E., Langston, A. A., Shulman, H., Sullivan, K. M., Lee, J., Henstorf, G., Storb, R. and Furst, D. E. (2006), Allogeneic marrow transplantation in patients with severe systemic sclerosis: Resolution of dermal fibrosis. Arthritis & Rheumatism, 54: 1982–1986. doi: 10.1002/art.21908
- Issue online: 26 MAY 2006
- Version of Record online: 26 MAY 2006
- Manuscript Received: 10 NOV 2005
- Manuscript Accepted: 9 MAR 2005
- NIH. Grant Numbers: HL-36444, AI-05419, CA-15704
To evaluate the safety and efficacy of allogeneic hematopoietic cell transplantation (HCT) after myeloablative conditioning in patients with severe systemic sclerosis (SSc).
Eligibility criteria for the study included SSc patients with features indicative of a poor prognosis. The myeloablative conditioning regimen included busulfan, cyclophosphamide, and antithymocyte globulin. Prophylaxis for graft-versus-host disease (GVHD) consisted of cyclosporine and methotrexate. Bone marrow was transplanted from HLA-identical siblings.
Two patients with diffuse cutaneous SSc and lung involvement who were refractory to conventional immunosuppressive treatment were enrolled in the study. In patient 1, there were no complications related to the conditioning regimen, and GVHD did not develop after transplantation. At 5 years after HCT, there was nearly complete resolution of the scleroderma and marked improvement in physical functioning. Internal organ function improved (lung) or remained stable. On examination of serial skin biopsy samples, there was resolution of the dermal fibrosis. Patient 2 experienced skin toxicity from the conditioning regimen and hypertensive crisis that was likely related to high-dose corticosteroids given for treatment of GVHD. Although this patient experienced an improvement in scleroderma and overall functioning, a fatal opportunistic infection developed 17 months after HCT.
Allogeneic HCT may result in sustained remission of SSc. GVHD and opportunistic infections are the major risks associated with allogeneic HCT for SSc, as for allogeneic HCT in general.