Dose-reduced versus standard conditioning followed by allogeneic stem-cell transplantation for patients with myelodysplastic syndrome: A prospective randomized phase III study of the EBMT (RICMAC Trial)

Nicolaus Kröger, Simona Iacobelli, Georg Nikolaus Franke, Uwe Platzbecker, Ruzena Uddin, Kai Hübel, Christof Scheid, Thomas Weber, Marie Robin, Matthias Stelljes, Boris Afanasyev, Dominik Heim, Giorgio Lambertenghi Deliliers, Francesco Onida, Peter Dreger, Massimo Pini, Stefano Guidi, Liisa Volin, Andreas Günther, Wolfgang BethgeXavier Poiré, Guido Kobbe, Marleen Van Os, Ronald Brand, Theo De Witte

Research output: Contribution to journalArticlepeer-review

Abstract

Purpose To compare a reduced-intensity conditioning regimen (RIC) with a myeloablative conditioning regimen (MAC) before allogeneic transplantation in patients with myelodysplastic syndrome (MDS) within a randomized trial. Patients and Methods Within the European Society of Blood and Marrow Transplantation, we conducted a prospective, multicenter, open-label, randomized phase III trial that compared a busulfan-based RIC with MAC in patients with MDS or secondary acute myeloid leukemia. A total of 129 patients were enrolled from 18 centers. Patientswere randomly assigned in a 1:1 ratio andwere stratified according to donor, age, and blast count. Results Engraftment was comparable between both groups. The CI of acute graft-versus-host disease II to IV was 32.3% after RIC and 37.5% after MAC (P = .35). The CI of chronic graft-versus-host disease was 61.6% after RIC and 64.7% after MAC (P = .76). The CI of nonrelapse mortality after 1 year was 17% (95% CI, 8% to 26%) after RIC and 25% (95% CI, 15% to 36%) after MAC (P = .29). The CI of relapse at 2 years was 17% (95% CI, 8% to 26%) after RIC and 15% (95% CI, 6% to 24%) after MAC (P = .6), which resulted in a 2-year relapse-free survival and overall survival of 62% (95% CI, 50% to 74%) and 76% (95% CI, 66% to 87%), respectively, after RIC, and 58% (95% CI, 46% to 71%) and 63% (95% CI, 51% to 75%), respectively, after MAC (P = .58 and P = .08, respectively). Conclusion This prospective, randomized trial of the European Society of Blood and Marrow Transplantation provides evidence that RIC resulted in at least a 2-year relapse-free survival and overall survival similar to MAC in patients with MDS or secondary acute myeloid leukemia.

Original languageEnglish
Pages (from-to)2157-2164
Number of pages8
JournalJournal of Clinical Oncology
Volume35
Issue number19
DOIs
Publication statusPublished - Jul 1 2017

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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