Results from the SCOT trial of hematopoietic stem cell therapy (HSCT) for scleroderma
The much-anticipated results of the SCOT trial of
hematopoietic stem cell therapy (HSCT) for scleroderma were released recently at the annual meeting of the American College of Rheumatology (ACR)
meeting in Washington, D.C.
The multicenter trial
compared the benefit and safety of intravenous (IV) cyclophosphamide to hematopoietic stem cell therapy (HSCT) for patients with aggressive
scleroderma. In a randomized controlled trial, patients treated with HSCT had
improved skin score and better event-free survival
compared to those treated with monthly IV cyclophosphamide. Serious
adverse effects were more frequent in the HSCT group.
At 54 months, HSCT
treated patients had 91 percent survival compared to 77 percent in cyclophosphamide
treated patients. These encouraging results
suggest that HSCT may be a treatment option for a carefully selected
group of patients who have serious scleroderma and no contraindications for
In light of the serious adverse effects and mortality associated
in some cases with HSCT, patients should be carefully
evaluated by physicians experienced with scleroderma and HSCT to
determine whether this treatment option is appropriate for them.
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