Autologous Stem-Cell Transplantation vs. Cyclophosphamide in Patients with Severe Scleroderma

Patients with diffuse scleroderma often have poor prognoses. Therapies generally have targeted specific organ involvement (e.g., angiotensin-converting–enzyme inhibitors for renal crisis). Cyclophosphamide and autologous stem-cell transplantation often are successful in improving skin sclerosis and pulmonary function, although safety and long-term efficacy are a concern. In this open-label, phase 2 trial, U.S. researchers randomized 75 patients with scleroderma and pulmonary or renal involvement to receive 12 monthly cyclophosphamide infusions or myeloablative autologous stem-cell transplantation.

In per-protocol analyses, event-free survival (i.e., survival without respiratory, renal, or cardiac failure) was significantly higher in the tran…