Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial

JM Van Laar, D Farge, JK Sont, K Naraghi… - Jama, 2014 - jamanetwork.com
Importance High-dose immunosuppressive therapy and autologous hematopoietic stem cell
transplantation (HSCT) have shown efficacy in systemic sclerosis in phase 1 and small
phase 2 trials. Objective To compare efficacy and safety of HSCT vs 12 successive monthly
intravenous pulses of cyclophosphamide. Design, Setting, and Participants The Autologous
Stem Cell Transplantation International Scleroderma (ASTIS) trial, a phase 3, multicenter,
randomized (1: 1), open-label, parallel-group, clinical trial conducted in 10 countries at 29 …