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Blood, 15 August 2007, Vol. 110, No. 4, pp. 1388-1396. Prepublished online as a Blood First Edition Paper on April 23, 2007; DOI 10.1182/blood-2007-02-072389. This Article Full Text Full Text (PDF) All Versions of this Article: blood-2007-02-072389v1 110/4/1388    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Nash, R. A. Articles by Furst, D. E. Search for Related Content PubMed PubMed Citation Articles by Nash, R. A. Articles by Furst, D. E. Related Collections Transplantation Immunotherapy Clinical Trials and Observations Related Article in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  TRANSPLANTATION High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study Richard A. Nash1,2, Peter A. McSweeney3, Leslie J. Crofford4, Muneer Abidi5, Chien-Shing Chen6, J. David Godwin2, Theodore A. Gooley1,2, Leona Holmberg1,2, Gretchen Henstorf1, C. Fred LeMaistre7, Maureen D. Mayes8, Kevin T. McDonagh4, Bernadette McLaughlin1, Jerry A. Molitor9, J. Lee Nelson1,2, Howard Shulman1,2, Rainer Storb1,2, Federico Viganego1, Mark H. Wener2, James R. Seibold10, Keith M. Sullivan11, and Daniel E. Furst12 1 Fred Hutchinson Cancer Research Center, Seattle, WA; 2 University of Washington, Seattle; 3 Rocky Mountain Cancer Center, Denver, CO; 4 University of Kentucky, Lexington; 5 Wayne State University, Detroit, MI; 6 Loma Linda University, CA; 7 Texas Transplant Institute, San Antonio; 8 University of Texas Health Science Center, Houston; 9 Virginia-Mason Medical Center, Seattle, WA; 10 University of Michigan, Ann Arbor; 11 Duke University Medical Center, Durham, NC; and 12 University of California, Los Angeles More effective therapeutic strategies are required for patients with poor-prognosis systemic sclerosis (SSc). A phase 2 single-arm study of high-dose immunosuppressive therapy (HDIT) and autologous CD34-selected hematopoietic cell transplantation (HCT) was conducted in 34 patients with diffuse cutaneous SSc. HDIT included total body irradiation (800 cGy) with lung shielding, cyclophosphamide (120 mg/kg), and equine antithymocyte globulin (90 mg/kg). Neutrophil and platelet counts were recovered by 9 (range, 7 to 13) and 11 (range, 7 to 25) days after HCT, respectively. Seventeen of 27 (63%) evaluable patients who survived at least 1 year after HDIT had sustained responses at a median follow-up of 4 (range, 1 to 8) years. There was a major improvement in skin (modified Rodnan skin score, –22.08; P < .001) and overall function (modified Health Assessment Questionnaire Disability Index, –1.03; P < .001) at final evaluation. Importantly, for the first time, biopsies confirmed a statistically significant decrease of dermal fibrosis compared with baseline (P < .001). Lung, heart, and kidney function, in general, remained clinically stable. There were 12 deaths during the study (transplantation-related, 8; SSc-related, 4). The estimated progression-free survival was 64% at 5 years. Sustained responses including a decrease in dermal fibrosis were observed exceeding those previously reported with other therapies. HDIT and autologous HCT for SSc should be evaluated in a randomized clinical trial. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Article in Blood Online: Autologous HSCT: toward scleroderma treatment Franck Verrecchia and Dominique Farge Blood 2007 110: 1088-1089. [Full Text] [PDF] This article has been cited by other articles: R. K. Burt, Y. Loh, W. Pearce, N. Beohar, W. G. Barr, R. Craig, Y. Wen, J. A. Rapp, and J. Kessler Clinical Applications of Blood-Derived and Marrow-Derived Stem Cells for Nonmalignant Diseases JAMA, February 27, 2008; 299(8): 925 - 936. [Abstract] [Full Text] [PDF]

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