title: Impact of achievement of complete cytogenetic response on outcome in patients with myelodysplastic syndromes treated with hypomethylating agents.
abstract: Two hundred and sixteen consecutive patients with MDS and abnormal karyotype treated with hypomethylating agents between 4/04 and 10/12 were reviewed. Median follow-up was 17 months. Using IWG criteria, best responses were complete response (CR) in 79 patients (37%), partial response (PR) in 4 (2%), and hematologic improvement (HI) in 10 (5%). Cytogenetic response (CyR) was achieved in 78 patients (36%): complete (CCyR) in 62 (29%) and partial in 16 (7%). CyR was achieved in 48 of 79 patients (61%) with CR, 1 of 14 (7%) with PR/HI, and in 29 of the 123 (24%) with no morphologic response. Median overall survival (OS) and leukemia-free survival (LFS) for patients with and without CCyR were 21 and 13 months (P = .007), and 16 and 9 months (P = .001), respectively. By multivariate analysis, the achievement of CCyR was predictive for better OS (HR = 2.1; P < .001). In conclusion, CyR occurs at a rate of 36% (complete in 29%) in patients with MDS treated with HMA and is not always associated with morphological response. The achievement of CCyR is associated with survival improvement and constitutes a major predictive factor for outcome particularly in patients without morphologic response. Therefore, the achievement of CCyR should be considered a milestone in the management of patients with MDS.
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Elias
authors.identifier: http://orcid.org/0000-0003-4465-6119
authors.initials: E
authors.lastname: Jabbour
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Paolo
authors.identifier: http://orcid.org/0000-0002-7445-1459
authors.initials: P
authors.lastname: Strati
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Monica
authors.initials: M
authors.lastname: Cabrero
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Susan
authors.initials: S
authors.lastname: O'Brien
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Farhad
authors.initials: F
authors.lastname: Ravandi
authors.affiliation: Department of Hematopathology, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Carlos
authors.initials: C
authors.lastname: Bueso-Ramos
authors.affiliation: Department of Biostatistics, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Qiao
authors.initials: Q
authors.lastname: Wei
authors.affiliation: Department of Biostatistics, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Jianhua
authors.initials: J
authors.lastname: Hu
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Simon
authors.initials: S
authors.lastname: Abi Aad
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Nicholas J
authors.identifier: http://orcid.org/0000-0002-2983-2738
authors.initials: NJ
authors.lastname: Short
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Courtney
authors.initials: C
authors.lastname: Dinardo
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Naval
authors.initials: N
authors.lastname: Daver
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Tapan
authors.initials: T
authors.lastname: Kadia
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: William
authors.initials: W
authors.lastname: Wierda
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Yue
authors.initials: Y
authors.lastname: Wei
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Simona
authors.initials: S
authors.lastname: Colla
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Gautam
authors.initials: G
authors.lastname: Borthakur
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Zeev
authors.initials: Z
authors.lastname: Estrov
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Hagop
authors.initials: H
authors.lastname: Kantarjian
authors.affiliation: Department of Leukemia, University of Texas M.D. Anderson Cancer Center, Houston, Texas, 77030, USA.
authors.forename: Guillermo
authors.initials: G
authors.lastname: Garcia-Manero
mesh_terms: D059786:Abnormal Karyotype; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020732:Cytogenetic Analysis; D019175:DNA Methylation; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality; D012074:Remission Induction / Q000379:methods; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome; D055815:Young Adult