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Blood, 1 September 2002, Vol. 100, No. 5, pp. 1602-1610
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
High-dose immunosuppressive therapy for severe systemic
sclerosis: initial outcomes
Peter A. McSweeney,
Richard
A. Nash,
Keith M. Sullivan,
Jan Storek,
Leslie J. Crofford,
Roger Dansey,
Maureen D. Mayes,
Kevin T. McDonagh,
J. Lee Nelson,
Theodore A. Gooley,
Leona A. Holmberg,
C. S. Chen,
Mark H. Wener,
Katherine Ryan,
Julie Sunderhaus,
Ken Russell,
John Rambharose,
Rainer Storb, and
Daniel E. Furst
From the University of Colorado Health Sciences Center,
Denver; Fred Hutchinson Cancer Research Center, University of
Washington School of Medicine, and Virginia Mason Medical Center;
Seattle, WA; Duke University, Durham, NC; Loma Linda University, CA;
University of Michigan, Ann Arbor; Karmanos Cancer Institute/Wayne
State University, Detroit, MI.
Systemic sclerosis (SSc) is a multisystem disease of presumed
autoimmune pathogenesis for which no proven effective treatment exists.
High-dose immunosuppressive therapy (HDIT) has been proposed as an
investigational treatment for severe autoimmune diseases. Nineteen
patients with poor-prognosis SSc underwent HDIT. The median age was 40 years (range, 23-61 years), the median modified Rodnan skin score (a
measure of dermal sclerosis) was 31, and the median DLCO was 57%.
Conditioning therapy involved 800 cGy total body irradiation (TBI) (± lung shielding to approximately 200 cGy), 120 mg/kg cyclophosphamide,
and 90 mg/kg equine antithymocyte globulin. CD34-selected
granulocyte-colony-stimulating factor-mobilized autologous blood stem
cells provided hematopoietic rescue. With median follow-up at 14.7 months, the Kaplan-Meier estimated 2-year survival rate was 79%. Three
patients died of treatment complications and one of disease
progression. Two of the first 8 patients had fatal regimen-related
pulmonary injury, a complication not found among 11 subsequent patients
who received lung shielding for TBI. Overall, internal organ functions
were stable to slightly worse after HDIT, and 4 patients had
progressive or nonresponsive disease. As measured by modified Rodnan
skin scores and modified health assessment questionnaire disability
index (mHAQ-DI) scores, significant disease responses occurred in 12 of
12 patients evaluated at 1 year after HDIT. In conclusion, though
important treatment-related toxicities occurred after HDIT for SSc,
modifications of initial approaches appear to reduce treatment risks.
Responses in skin and mHAQ-DI scores exceed those reported with other
therapies, suggesting that HDIT is a promising new therapy for SSc that
should be evaluated in prospective randomized studies.
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