Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma
Status:
Completed
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
Scleroderma is a systemic disorder categorized as an immunologically mediated disease that
causes collagen deposition of skin and visceral organs. The molecular pathogenesis of
scleroderma has been elusive, although vasculopathy and immune mediated mechanisms are
thought to be important. Once extensive cutaneous or visceral disease occurs, prognosis is
significantly shorter than the general population. Although various treatments have been
tried, none of them seems to have changed the natural history of scleroderma. Standard dose
immunosuppressive treatment has been disappointing. Recently, cyclophosphamide at 1-2
mg/kg/day orally or 800-1400 mg intravenous (IV) monthly for 6-9 months has proven effective
in treatment of scleroderma alveolitis (1). Recent phase I studies of immunoablation with
autologous peripheral blood stem cell transplantation (PBSCT) showed some promising data, but
the exact efficacy is undetermined (2,3). We now propose, as a phase II randomized study,
autologous unmanipulated PBSCT versus pulse cyclophosphamide in patients with systemic
scleroderma.