Open AccessAllogeneic marrow transplantation in patients with severe systemic sclerosis: Resolution of dermal fibrosis
Author(s) -
Nash Richard A.,
McSweeney Peter A.,
Nelson J. Lee,
Wener Mark,
Georges George E.,
Langston Amelia A.,
Shulman Howard,
Sullivan Keith M.,
Lee Julie,
Henstorf Gretchen,
Storb Rainer,
Furst Daniel E.
Publication year - 2006
Publication title -
arthritis & rheumatism
Language(s) - English
Resource type - Journals
eISSN - 1529-0131
pISSN - 0004-3591
DOI - 10.1002/art.21908
Subject(s) - medicine , busulfan , cyclophosphamide , scleroderma (fungus) , transplantation , graft versus host disease , regimen , methotrexate , surgery , gastroenterology , fibrosis , hematopoietic stem cell transplantation , immunology , chemotherapy , inoculation
Objective To evaluate the safety and efficacy of allogeneic hematopoietic cell transplantation (HCT) after myeloablative conditioning in patients with severe systemic sclerosis (SSc). Methods 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. Results 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. Conclusion 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.