title: De novo acute myeloid leukemia with 20-29% blasts is less aggressive than acute myeloid leukemia with ≥30% blasts in older adults: a Bone Marrow Pathology Group study.
abstract: It is controversial whether acute myeloid leukemia (AML) patients with 20-29% bone marrow (BM) blasts, formerly referred to as refractory anemia with excess blasts in transformation (RAEBT), should be considered AML or myelodysplastic syndrome (MDS) for the purposes of treatment and prognostication. We retrospectively studied 571 de novo AML in patients aged >50 years, including 142 RAEBT and 429 with ≥30% blasts (AML30), as well as 151 patients with 10-19% BM blasts (RAEB2). RAEBT patients were older and had lower white blood count, but higher hemoglobin, platelet count, and karyotype risk scores compared to AML30, while these features were similar to RAEB2. FLT3 and NPM1 mutations and monocytic morphology occurred more commonly in AML30 than in RAEBT. RAEBT patients were treated less often with induction therapy than AML30, whereas allogeneic stem cell transplant frequency was similar. The median and 4-year OS of RAEBT patients were longer than those of AML30 patients (20.5 vs 12.0 months and 28.6% vs 20.4%, respectively, P = 0.003); this difference in OS was manifested in patients in the intermediate UKMRC karyotype risk group, whereas OS of RAEBT patients and AML30 patients in the adverse karyotype risk group were not significantly different. Multivariable analysis showed that RAEBT (P < 0.0001), hemoglobin (P = 0.005), UKMRC karyotype risk group (P = 0.002), normal BM karyotype (P = 0.004), treatment with induction therapy (P < 0.0001), and stem cell transplant (P < 0.0001) were associated with longer OS. Our findings favor considering de novo RAEBT as a favorable prognostic subgroup of AML.
authors.affiliation: Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Robert Paul
authors.initials: RP
authors.lastname: Hasserjian
authors.forename: Federico
authors.initials: F
authors.lastname: Campigotto
authors.forename: Veronica
authors.initials: V
authors.lastname: Klepeis
authors.forename: Bin
authors.initials: B
authors.lastname: Fu
authors.forename: Sa A
authors.initials: SA
authors.lastname: Wang
authors.forename: Carlos
authors.initials: C
authors.lastname: Bueso-Ramos
authors.forename: Michael Joseph
authors.initials: MJ
authors.lastname: Cascio
authors.forename: Heesun Joyce
authors.initials: HJ
authors.lastname: Rogers
authors.forename: Eric Darryl
authors.initials: ED
authors.lastname: Hsi
authors.forename: Craig
authors.initials: C
authors.lastname: Soderquist
authors.forename: Adam
authors.initials: A
authors.lastname: Bagg
authors.forename: Jiong
authors.initials: J
authors.lastname: Yan
authors.forename: Rachel
authors.initials: R
authors.lastname: Ochs
authors.forename: Attilio
authors.initials: A
authors.lastname: Orazi
authors.forename: Frank
authors.initials: F
authors.lastname: Moore
authors.forename: Amer
authors.initials: A
authors.lastname: Mahmoud
authors.forename: Tracy Irene
authors.initials: TI
authors.lastname: George
authors.forename: Kathryn
authors.initials: K
authors.lastname: Foucar
authors.forename: Jamie
authors.initials: J
authors.lastname: Odem
authors.forename: Cassie
authors.initials: C
authors.lastname: Booth
authors.forename: William
authors.initials: W
authors.lastname: Morice
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: DeAngelo
authors.forename: David
authors.initials: D
authors.lastname: Steensma
authors.forename: Richard Maury
authors.initials: RM
authors.lastname: Stone
authors.forename: Donna
authors.initials: D
authors.lastname: Neuberg
authors.forename: Daniel Alan
authors.initials: DA
authors.lastname: Arber
mesh_terms: D000367:Age Factors; D000368:Aged; D000369:Aged, 80 and over; D000754:Anemia, Refractory, with Excess of Blasts / Q000235:genetics / Q000401:mortality / Q000473:pathology* / Q000628:therapy; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D001853:Bone Marrow / Q000473:pathology*; D002452:Cell Count; D002869:Chromosome Aberrations; D019175:DNA Methylation / Q000187:drug effects; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D053208:Kaplan-Meier Estimate; D059785:Karyotype; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality / Q000473:pathology* / Q000628:therapy; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009363:Neoplasm Proteins / Q000235:genetics; D014411:Neoplastic Stem Cells / Q000473:pathology; D009687:Nuclear Proteins / Q000235:genetics; D000090243:Nucleophosmin; D011379:Prognosis; D012074:Remission Induction; D012189:Retrospective Studies; D016896:Treatment Outcome; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics
chemical_list: C000716967:NPM1 protein, human; D009363:Neoplasm Proteins; D009687:Nuclear Proteins; D000090243:Nucleophosmin; C497970:FLT3 protein, human; D051941:fms-Like Tyrosine Kinase 3