title: Allogeneic hematopoietic cell transplantation in patients age 60-70 years with de novo high-risk myelodysplastic syndrome or secondary acute myelogenous leukemia: comparison with patients lacking donors who received azacitidine.
abstract: Standard 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.
authors.affiliation: Medizinische Klinik und Poliklinik I, Universitätsklinikum Carl Gustav Carus, Dresden, Germany. Uwe.Platzbecker@uniklinikum-dresden.de
authors.forename: Uwe
authors.initials: U
authors.lastname: Platzbecker
authors.forename: Johannes
authors.initials: J
authors.lastname: Schetelig
authors.forename: Jürgen
authors.initials: J
authors.lastname: Finke
authors.forename: Rudolf
authors.initials: R
authors.lastname: Trenschel
authors.forename: Bart L
authors.initials: BL
authors.lastname: Scott
authors.forename: Guido
authors.initials: G
authors.lastname: Kobbe
authors.forename: Kerstin
authors.initials: K
authors.lastname: Schaefer-Eckart
authors.forename: Martin
authors.initials: M
authors.lastname: Bornhäuser
authors.forename: Raphael
authors.initials: R
authors.lastname: Itzykson
authors.forename: Ulrich
authors.initials: U
authors.lastname: Germing
authors.forename: Dietrich
authors.initials: D
authors.lastname: Beelen
authors.forename: Gerhard
authors.initials: G
authors.lastname: Ehninger
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.forename: H Joachim
authors.initials: HJ
authors.lastname: Deeg
authors.forename: Lionel
authors.initials: L
authors.lastname: Adès
mesh_terms: D000368:Aged; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D009190:Myelodysplastic Syndromes / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D012189:Retrospective Studies; D012306:Risk; D016019:Survival Analysis; D014019:Tissue Donors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine