|
|
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 | Next Article 
Submitted February 6, 2007
Accepted April 16, 2007
High-dose immunosuppressive therapy and autologous
hematopoietic cell transplantation for severe systemic
sclerosis: long-term follow-up of the U.S. multicenter
pilot study
Richard A. Nash*, Peter A. McSweeney, Leslie J Crofford, Muneer Abidi, Chien-Shing Chen, J. David Godwin, Theodore A. Gooley, Leona A. Holmberg, Gretchen Henstorf, C. Fred LeMaistre, Maureen D Mayes, Kevin T McDonagh, Bernadette McLaughlin, Jerry A Molitor, J. Lee Nelson, Howard Shulman, Rainer Storb, Federico Viganego, Mark H Wener, James R Seibold, Keith M. Sullivan, and Daniel E Furst
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, United States
Bone Marrow Tansplantation Service, Rocky Mountain Cancer Center, Denver, CO, United States
Bone Marrow Transplantation Service, University of Kentucky, Lexington, KY, United States
Bone Marrow Transplantation Service, Wayne State University, Detroit, MI, United States
Bone Marrow Tansplantation Service, Loma Linda University, Loma Linda, CA, United States
Department of Radiology, University of Washington, Seattle, WA, United States
South Texas Cancer Institute, Texas Transplant Inst., San Antonio, TX, United States
Bone Marrow Tansplantation Service, University of Texas Health Science Center, Houston, TX, United States
Bone Marrow Tansplantation Service, Virginia Mason Medical Center, Seattle, WA, United States
Department of Rheumatology, University of Washington, Seattle, WA, United States
Department of Internal Medicine, University of Michigan, Ann Arbor, MI, United States
Department of Medicine, Duke University Medical Center, Durham, NC, United States
Department of Rheumatology, University of California, Los Angeles, CA, United States
* Corresponding author; email: rnash{at}fhcrc.org.
More effective therapeutic strategies are required for patients with poor-prognosis systemic sclerosis (SSc). A phase II, 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 recovered by 9 (7-13) and 11 (7-25) 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 (1-8) years. There was a major improvement in skin (mRSS: -22.08, P<.0001)and overall function (HAQ-DI: -1.03, P<.0001) at final evaluation. Importantly, for the first time, biopsies confirmed a statistically significant decrease of dermal fibrosis compared to baseline (P<0.0001). Lung, heart and kidney function, in general, remained clinically stable. There were 12 deaths during the study (transplant-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:
|
|
|
|
 
D. Daoussis, S.-N. C. Liossis, A. C. Tsamandas, C. Kalogeropoulou, A. Kazantzi, C. Sirinian, M. Karampetsou, G. Yiannopoulos, and A. P. Andonopoulos
Experience with rituximab in scleroderma: results from a 1-year, proof-of-principle study
Rheumatology,
September 15, 2009;
(2009)
kep093v2.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
D. LAUNAY, Z. MARJANOVIC, C. de BAZELAIRE, L. FLOREA, S. ZOHAR, H. KESHTMAND, C. DELIGNY, A. de RAIGNIAC, A. U. WELLS, and D. FARGE
Autologous Hematopoietic Stem Cell Transplant in Systemic Sclerosis: Quantitative High Resolution Computed Tomography of the Chest Scoring
J Rheumatol,
July 1, 2009;
36(7):
1460 - 1463.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
T. BOHGAKI, T. ATSUMI, M. BOHGAKI, A. FURUSAKI, M. KONDO, K. C. SATO-MATSUMURA, R. ABE, H. KATAOKA, T. HORITA, S. YASUDA, et al.
Immunological Reconstitution after Autologous Hematopoietic Stem Cell Transplantation in Patients with Systemic Sclerosis: Relationship Between Clinical Benefits and Intensity of Immunosuppression
J Rheumatol,
June 1, 2009;
36(6):
1240 - 1248.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
I. Sabnani, M. J. Zucker, E. D. Rosenstein, D. A. Baran, L. H. Arroyo, P. Tsang, M. Zubair, and V. Rivera
A novel therapeutic approach to the treatment of scleroderma-associated pulmonary complications: safety and efficacy of combination therapy with imatinib and cyclophosphamide
Rheumatology,
January 1, 2009;
48(1):
49 - 52.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J M van Laar, D Farge, and A Tyndall
Stem cell transplantation: a treatment option for severe systemic sclerosis?
Ann Rheum Dis,
December 1, 2008;
67(Suppl_3):
iii35 - iii38.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. R. Seibold, A. Tyndall, and D. E. Furst
Trial design: how must we move ahead?
Rheumatology,
October 1, 2008;
47(suppl_5):
v57 - v58.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
F. Braiteh, S. R. Hymes, S. A. Giralt, and R. Jones
Complete Remission of Psoriasis After Autologous Hematopoietic Stem-Cell Transplantation for Multiple Myeloma
J. Clin. Oncol.,
September 20, 2008;
26(27):
4511 - 4513.
[Full Text]
[PDF]
|
|
|
|
|
|
|
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]
|
|
|
|
|
