title: Azacitidine in patients with WHO-defined AML - results of 155 patients from the Austrian Azacitidine Registry of the AGMT-Study Group.
abstract: OBJECTIVE
  The Austrian Azacitidine Registry is a multi-center database (ClinicalTrials.gov: NCT01595295). The nature and intent of the registry was to gain a comprehensive view of the use, safety and efficacy of the drug in a broad range of AML-patients treated in real-life scenarios.
  
  
  METHODS
  The sole inclusion criteria were the diagnosis of WHO-AML and treatment with at least one dose of azacitidine. No formal exclusion criteria existed. A total of 155 AML-patients who were mostly unfit/ineligible for intensive chemotherapy, or had progressed despite conventional treatment, were included. True ITT-analyses and exploratory analyses regarding the potential prognostic value of baseline-variables/performance-/comorbidity-/risk-scores on overall survival (OS), were performed.
  
  
  RESULTS
  In this cohort of 155 pretreated (60%), and/or comorbid (87%), elderly (45% ≥75 years) AML-patients, azacitidine was well tolerated and efficacious, with an overall response rate (CR, mCR, PR, HI) of 45% in the total cohort (ITT) and 65% in patients evaluable according to IWG-criteria, respectively. Pre-treatment with conventional chemotherapy (P = .113), age ≤/>80 years (P = .853), number of comorbidities (P = .476), and bone marrow (BM) blast count (P = .663) did not influence OS. In multivariate analysis hematologic improvement alone (without the requirement of concomitant bone marrow blast reduction), although currently not regarded as a standard form of response assessment in AML, was sufficient to confer OS benefit (18.9 vs. 6.0 months; P = .0015). Further deepening of response after first response was associated with improved OS (24.7 vs. 13.7 months; P < .001).
  
  
  CONCLUSIONS
  In this large cohort of AML-patients treated with azacitidine, age >80 years, number of comorbidities and/or BM-blasts >30% did not adversely impact OS.
authors.forename: Lisa
authors.initials: L
authors.lastname: Pleyer
authors.forename: Reinhard
authors.initials: R
authors.lastname: Stauder
authors.forename: Sonja
authors.initials: S
authors.lastname: Burgstaller
authors.forename: Martin
authors.initials: M
authors.lastname: Schreder
authors.forename: Christoph
authors.initials: C
authors.lastname: Tinchon
authors.forename: Michael
authors.initials: M
authors.lastname: Pfeilstocker
authors.forename: Susanne
authors.initials: S
authors.lastname: Steinkirchner
authors.forename: Thomas
authors.initials: T
authors.lastname: Melchardt
authors.forename: Martina
authors.initials: M
authors.lastname: Mitrovic
authors.forename: Michael
authors.initials: M
authors.lastname: Girschikofsky
authors.forename: Alois
authors.initials: A
authors.lastname: Lang
authors.forename: Peter
authors.initials: P
authors.lastname: Krippl
authors.forename: Thamer
authors.initials: T
authors.lastname: Sliwa
authors.forename: Alexander
authors.initials: A
authors.lastname: Egle
authors.forename: Werner
authors.initials: W
authors.lastname: Linkesch
authors.forename: Daniela
authors.initials: D
authors.lastname: Voskova
authors.forename: Hubert
authors.initials: H
authors.lastname: Angermann
authors.forename: Richard
authors.initials: R
authors.lastname: Greil
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001317:Austria; D001374:Azacitidine / Q000009:adverse effects / Q000627:therapeutic use*; D015331:Cohort Studies; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012042:Registries; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine