title: Long-term follow-up of cytogenetically normal CEBPA-mutated AML.
abstract: BACKGROUND
  The aim of this study was to analyze the long-term survival of AML patients with CEBPA mutations.
  
  
  METHODS
  We investigated 88 AML patients with a median age of 61 years and (1) cytogenetically normal AML (CN-AML), (2) monoallelic (moCEBPA) or biallelic (biCEBPA) CEBPA mutation, and (3) intensive induction treatment. 60/88 patients have been described previously with a shorter follow-up.
  
  
  RESULTS
  Median follow-up time was 9.8 years (95% CI: 9.4-10.1 years) compared to 3.2 and 5.2 years in our former analyses. Patients with biCEBPA mutations survived significantly longer compared to those with moCEBPA (median overall survival (OS) 9.6 years vs. 1.7 years, p = 0.008). Patients ≤ 60 years and biCEBPA mutations showed a favorable prognosis with a 10-year OS rate of 81%.
  
  
  CONCLUSIONS
  In this long-term observation we confirmed the favorable prognostic outcome of patients with biCEBPA mutations compared to moCEBPA-mutated CN-AML. The high probability of OS (81%) in younger patients is helpful to guide intensity of postremission therapy.
authors.forename: Friederike
authors.initials: F
authors.lastname: Pastore
authors.forename: Daniela
authors.initials: D
authors.lastname: Kling
authors.forename: Eva
authors.initials: E
authors.lastname: Hoster
authors.forename: Annika
authors.initials: A
authors.lastname: Dufour
authors.forename: Nikola P
authors.initials: NP
authors.lastname: Konstandin
authors.forename: Stephanie
authors.initials: S
authors.lastname: Schneider
authors.forename: Maria C
authors.initials: MC
authors.lastname: Sauerland
authors.forename: Wolfgang E
authors.initials: WE
authors.lastname: Berdel
authors.forename: Thomas
authors.initials: T
authors.lastname: Buechner
authors.forename: Bernhard
authors.initials: B
authors.lastname: Woermann
authors.forename: Jan
authors.initials: J
authors.lastname: Braess
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Hiddemann
authors.forename: Karsten
authors.initials: K
authors.lastname: Spiekermann
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use; D014408:Biomarkers, Tumor / Q000235:genetics; D022762:CCAAT-Enhancer-Binding Proteins / Q000235:genetics*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D020869:Gene Expression Profiling; D006801:Humans; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy / Q000235:genetics* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D009154:Mutation; D011379:Prognosis; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor; D022762:CCAAT-Enhancer-Binding Proteins; C515848:CEBPA protein, human