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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.
 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.
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