Understanding Systemic Sclerosis (SSc) and SCOT
- Download the monograph, Systemic Sclerosis and the SCOT Study (pdf)
The Pilot Study
The Fred Hutchinson Cancer Research Center (FHCRC) Protocol 1019 is a multicenter, pilot study that formed a basis for the SCOT study. The regimen includes HDIT using total body irradiation (TBI) (800 cGY), cylophosphamide 120 mg/kg, and equine antithymocyte globulin (ATGAM) 90 mg/kg followed by autologous peripheral blood stem cell infusion.
Summary Outcome Data
Data from 33 participants enrolled between January 1997 and December 2003 with follow-up through May 2004 reveal:
- Significant improvement in skin assessments
- Significant improvement in the modified Health Assessment Questionnaires (HAQs)
- Stable forced vital capacity (FVC), diffusion in liters of carbon monoxide (DLCO), ejection fraction, and serum creatinine
- 5 patients died due to study-related reasons
- 5 patients died due to disease progression
- 4 patients are alive with progression
- 19 patients are alive without progression
Key Points
Data from the pilot study demonstrate impressive disease responses in severe SSc.
However, there are ongoing concerns regarding potential risks and side effects. Lessons learned from preliminary studies have influenced the design of the SCOT study. Enhanced study measures, modified study-procedure regimens, and close monitoring are expected to reduce risk.
The SCOT study offers a unique opportunity to conduct basic studies of the pathology of SSc. It is designed to determine whether HDIT plus autologous transplant is better than intervention with pulse cyclophosphamide for severe SSc.
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