Jacob M van Laar, Dominique Farge, Jacob K Sont, Kamran Naraghi, Zora Marjanovic, Jérôme Larghero, Annemie J Schuerwegh, Erik W A Marijt, Madelon C Vonk, Anton V Schattenberg, Marco Matucci-Cerinic, Alexandre E Voskuyl, Arjan A van de Loosdrecht, Thomas Daikeler, Ina Kötter, Marc Schmalzing, Thierry Martin, Bruno Lioure, Stefan M Weiner, Alexander Kreuter, Christophe Deligny, Jean-Marc Durand, Paul Emery, Klaus P Machold, Francoise Sarrot-Reynauld, Klaus Warnatz, Daniel F P Adoue, Joël Constans, Hans-Peter Tony, Nicoletta Del Papa, Athanasios Fassas, Andrea Himsel, David Launay, Andrea Lo Monaco, Pierre Philippe, Isabelle Quéré, Éric Rich, Rene Westhovens, Bridget Griffiths, Riccardo Saccardi, Frank H van den Hoogen, Willem E Fibbe, Gérard Socié, Alois Gratwohl, Alan Tyndall
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 centers with access to a European Group for Blood and Marrow Transplantation-registered transplant facility...
June 25, 2014: JAMA