title: Identification of molecular and cytogenetic risk factors for unfavorable core-binding factor-positive adult AML with post-remission treatment outcome analysis including transplantation.
abstract: Emerging molecular studies have identified a subgroup of patients with unfavorable core-binding factor-positive (CBF)-AML who should be treated by intensified post-remission treatments. We analyzed 264 adults with CBF-AML from 2002 to 2011, and focused on 206 patients who achieved CR after standard '3+7' induction chemotherapy. Patients who achieved CR with an available donor were treated with allogeneic hematopoietic SCT (allo-HSCT, n=115) and the rest were treated with autologous (auto) HSCT (n=72) or chemotherapy alone (n=19). OS was not significantly different between CBFβ/MYH11 (n=62) and RUNX1/RUNX1T1 (n=144), and auto-HSCT showed favorable OS compared with allo-HSCT or chemotherapy alone. Cytogenetic analysis identified that inv(16) without trisomy had a favorable OS and t(8;21) with additional chromosomes had an unfavorable OS, but multivariate analysis revealed those were NS. Patients with c-kit mutation showed inferior OS. For transplanted patients, residual post-transplant CBF-minimal residual disease quantitative PCR with higher WT1 expression at D+60 showed the worst OS with a higher incidence of relapse. Conclusively, we found that unfavorable CBF-AML can be defined with risk stratification using cytogenetic and molecular studies, and a careful risk-adapted treatment approach using frontline transplantation with novel therapies should be evaluated for this particular risk subgroup.
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: J-H
authors.initials: JH
authors.lastname: Yoon
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: H-J
authors.initials: HJ
authors.lastname: Kim
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: J-W
authors.initials: JW
authors.lastname: Kim
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Y-W
authors.initials: YW
authors.lastname: Jeon
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: S-H
authors.initials: SH
authors.lastname: Shin
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: S-E
authors.initials: SE
authors.lastname: Lee
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: B-S
authors.initials: BS
authors.lastname: Cho
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: K-S
authors.initials: KS
authors.lastname: Eom
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Y-J
authors.initials: YJ
authors.lastname: Kim
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: S
authors.initials: S
authors.lastname: Lee
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: C-K
authors.initials: CK
authors.lastname: Min
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: S-G
authors.initials: SG
authors.lastname: Cho
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: J-W
authors.initials: JW
authors.lastname: Lee
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: W-S
authors.initials: WS
authors.lastname: Min
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Department of Hematology, Cancer Research Institute, Seoul St Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: C-W
authors.initials: CW
authors.lastname: Park
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D007446:Chromosome Inversion; D050660:Core Binding Factors / Q000378:metabolism; D003582:Cytogenetics; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics* / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D009154:Mutation; D011379:Prognosis; D012008:Recurrence; D012074:Remission Induction; D012189:Retrospective Studies; D012307:Risk Factors; D014178:Translocation, Genetic; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents; D050660:Core Binding Factors
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