Nicolaus Kröger, University Medical Center Hamburg-Eppendorf, Hamburg; Georg-Nikolaus Franke, University Hospital Leipzig, Leipzig; Uwe Platzbecker, University Hospital Dresden, Dresden; Kai Hübel and Christof Scheid, University of Cologne, Cologne; Thomas Weber, University Hospital Halle (Saale), Martin-Luther-University Halle-Wittenberg, Halle; Matthias Stelljes, University of Münster, Münster; Peter Dreger, University of Heidelberg, Heidelberg; Andreas Günther, University Hospital Schleswig Holstein Campus Kiel, Kiel; Wolfgang Bethge, University Hospital Tübingen, Tübingen; Guido Kobbe, Heinrich Heine University, Düsseldorf, Germany; Simona Iacobelli, Università Tor Vergata, Rome; Giorgio Lambertenghi Deliliers and Francesco Onida, Fondazione IRCC Ca' Granda Ospedale Maggiore Policlinico, University of Milano, Milan; Massimo Pini, Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo, Alessandria; Stefano Guidi, Azienda Ospedaliera Universitaria Careggi, Florence, Italy; Ruzena Uddin, Guy's Hospital, London, United Kingdom; Marie Robin, Hôpital Saint Louis, Assistance Publique Hôpitaux Paris, France; Boris Afanasyev, SPB Pavlov Medical University, St Petersburg, Russia; Dominik Heim, University Hospital Basel, Basel Switzerland; Liisa Volin, Helsinki University Hospital, Helsiniki, Finland; Xavier Poiré, Cliniques Universitaires St-Luc, Brussels, Belgium; Marleen van Os, European Group of Blood and Marrow Transplantation Clinical Trials Office; Ronald Brand, Leiden University Medical Center, Leiden; and Theo de Witte, Radboud University Medical Center, Nijmegen, the Netherlands.
J Clin Oncol. 2017 Jul 1;35(19):2157-2164. doi: 10.1200/JCO.2016.70.7349. Epub 2017 May 2.
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. Patients were randomly assigned in a 1:1 ratio and were 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.
在一项随机试验中，比较骨髓增生异常综合征（MDS）患者异体移植前接受低强度预处理方案（RIC）与高强度预处理方案（MAC）的效果。
在欧洲血液和骨髓移植学会内，我们开展了一项前瞻性、多中心、开放标签、随机 III 期试验，比较了以白消安为基础的 RIC 与 MDS 或继发性急性髓系白血病患者的 MAC。共从 18 个中心招募了 129 例患者。患者以 1:1 的比例随机分组，并根据供体、年龄和原始细胞计数进行分层。
两组的植入情况相当。RIC 组和 MAC 组的 II 至 IV 级急性移植物抗宿主病累积发生率分别为 32.3%和 37.5%（P =.35）。RIC 组和 MAC 组慢性移植物抗宿主病的累积发生率分别为 61.6%和 64.7%（P =.76）。RIC 组和 MAC 组 1 年后非复发死亡率分别为 17%（95%CI，8%26%）和 25%（95%CI，15%36%）（P =.29）。RIC 组和 MAC 组 2 年复发率分别为 17%（95%CI，8%26%）和 15%（95%CI，6%24%）（P =.6），导致 RIC 组 2 年无复发生存率和总生存率分别为 62%（95%CI，50%74%）和 76%（95%CI，66%87%），MAC 组分别为 58%（95%CI，46%71%）和 63%（95%CI，51%75%）（P =.58 和 P =.08）。
这项由欧洲血液和骨髓移植学会开展的前瞻性随机试验提供了证据，表明对于 MDS 或继发性急性髓系白血病患者，RIC 可带来至少 2 年无复发生存率和总生存率，与 MAC 相似。
