• Uwe Platzbecker, Johannes Schetelig, Jürgen Finke, Rudolf Trenschel, Bart L Scott, Guido Kobbe, Kerstin Schaefer-Eckart, Martin Bornhäuser, Raphael Itzykson, Ulrich Germing, Dietrich Beelen, Gerhard Ehninger, Pierre Fenaux, H Joachim Deeg, Lionel Adès, German MDS Study, Cooperative Transplant Study Group, Fred Hutchinson Cancer Research Center, and Groupe Francophone des Myelodysplasies.
• Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus, Dresden, Germany. Uwe.Platzbecker@uniklinikum-dresden.de
• Biol. Blood Marrow Transplant. 2012 Sep 1; 18 (9): 1415-21.

AbstractStandard first-line therapy for older patients with high-risk myelodysplastic syndrome (MDS) includes hypomethylating agents, such as azacitidine (AZA). However, the only approach with curative potential remains allogeneic hematopoietic cell transplantation (HCT). To date, no direct comparison of both strategies has been reported. The outcomes of 2 well-balanced cohorts of patients with high-risk MDS defined by age (60-70 years), performance status (Eastern Cooperative Oncology Group score ≤2), and donor availability (yes/no) were compared, including 103 patients undergoing HCT and 75 patients without this option who received AZA. The estimated 2-year overall survival after the start of treatment was 39% (95% confidence interval, 30%-50%) for the patients undergoing HCT and 23% (95% confidence interval, 14%-40%) for the patients receiving AZA therapy. In a multivariate Cox regression analysis of all patients (n = 178), Eastern Cooperative Oncology Group score (0 versus 1 versus 2; hazard ratio [HR], 2.9/3.9; P <  .001), cytogenetics (good versus intermediate versus poor; HR, 1.2/1.7; P = .026), and treatment (HCT versus AZA; HR, 0.3; P = .007) were associated with overall survival. This retrospective cohort analysis suggests a survival advantage for allogeneic HCT compared with AZA therapy in medically fit patients with high-risk MDS age 60-70 years. Prospective controlled studies are warranted.Copyright © 2012 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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