title: The value of allogeneic and autologous hematopoietic stem cell transplantation in prognostically favorable acute myeloid leukemia with double mutant CEBPA.
abstract: The clinical value of allogeneic hematopoietic stem cell transplantation (alloHSCT) and autologous hematopoietic stem cell transplantation (autoHSCT) in the subtype of acute myeloid leukemia (AML) with double mutant CEBPA (CEBPAdm) has remained unsettled. Among 2983 patients analyzed for CEBPA mutational status (age 18-60 years) treated on 4 published Dutch-Belgian-Swiss Hemato-Oncology Cooperative Group (HOVON/SAKK) and 3 German-Austrian AML Study Group (AMLSG) protocols (2 published, 1 registered, clinicaltrials.gov NCT00151255), 124 had AML with CEBPAdm and achieved first complete remission (CR1). Evaluation of the clinical impact of alloHSCT and autoHSCT vs chemotherapy was performed by addressing time dependency in the statistical analyses. Thirty-two patients proceeded to alloHSCT from a matched related (MRD, n = 29) or a matched unrelated donor (MUD, n = 3), 20 to autoHSCT in CR1 and 72 received chemotherapy. Relapse-free survival was significantly superior in patients receiving an alloHSCT or autoHSCT in CR1 as compared with chemotherapy (P < .001), whereas overall survival was not different (P < .12). Forty-five patients relapsed. Of 42 patients treated with reinduction therapy, 35 achieved a second CR (83%) and most patients (n = 33) received an alloHSCT MRD, n = 11; MUD, n = 19; haplo-identical donor, n = 3). Survival of relapsed patients measured from date of relapse was 46% after 3 years. Adult AML patients with CEBPAdm benefit from alloHSCT and autoHSCT; relapsed patients still have a favorable outcome after reinduction followed by alloHSCT.
authors.affiliation: Department of Internal Medicine III, University of Ulm, Ulm, Germany. richard.schlenk@uniklinik-ulm.de
authors.forename: Richard F
authors.initials: RF
authors.lastname: Schlenk
authors.forename: Erdogan
authors.initials: E
authors.lastname: Taskesen
authors.forename: Yvette
authors.initials: Y
authors.lastname: van Norden
authors.forename: Jürgen
authors.initials: J
authors.lastname: Krauter
authors.forename: Arnold
authors.initials: A
authors.lastname: Ganser
authors.forename: Lars
authors.initials: L
authors.lastname: Bullinger
authors.forename: Verena I
authors.initials: VI
authors.lastname: Gaidzik
authors.forename: Peter
authors.initials: P
authors.lastname: Paschka
authors.forename: Andrea
authors.initials: A
authors.lastname: Corbacioglu
authors.forename: Gudrun
authors.initials: G
authors.lastname: Göhring
authors.forename: Andrea
authors.initials: A
authors.lastname: Kündgen
authors.forename: Gerhard
authors.initials: G
authors.lastname: Held
authors.forename: Katharina
authors.initials: K
authors.lastname: Götze
authors.forename: Edo
authors.initials: E
authors.lastname: Vellenga
authors.forename: Juergen
authors.initials: J
authors.lastname: Kuball
authors.forename: Urs
authors.initials: U
authors.lastname: Schanz
authors.forename: Jakob
authors.initials: J
authors.lastname: Passweg
authors.forename: Thomas
authors.initials: T
authors.lastname: Pabst
authors.forename: Johan
authors.initials: J
authors.lastname: Maertens
authors.forename: Gert J
authors.initials: GJ
authors.lastname: Ossenkoppele
authors.forename: Ruud
authors.initials: R
authors.lastname: Delwel
authors.forename: Hartmut
authors.initials: H
authors.lastname: Döhner
authors.forename: Jan J
authors.initials: JJ
authors.lastname: Cornelissen
authors.forename: Konstanze
authors.initials: K
authors.lastname: Döhner
authors.forename: Bob
authors.initials: B
authors.lastname: Löwenberg
mesh_terms: D000293:Adolescent; D000328:Adult; D022762:CCAAT-Enhancer-Binding Proteins / Q000235:genetics*; D015331:Cohort Studies; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics* / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009154:Mutation / Q000502:physiology; D011379:Prognosis; D012074:Remission Induction / Q000379:methods; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D022762:CCAAT-Enhancer-Binding Proteins; C515848:CEBPA protein, human