%0 Journal Article
%A Döhner, Hartmut
%A Weber, Daniela
%A Krzykalla, Julia
%A Fiedler, Walter
%A Kühn, Michael W M
%A Schroeder, Thomas
%A Mayer, Karin
%A Lübbert, Michael
%A Wattad, Mohammed
%A Götze, Katharina
%A Fransecky, Lars
%A Koller, Elisabeth
%A Wulf, Gerald
%A Schleicher, Jan
%A Ringhoffer, Mark
%A Greil, Richard
%A Hertenstein, Bernd
%A Krauter, Jürgen
%A Martens, Uwe M
%A Nachbaur, David
%A Samra, Maisun Abu
%A Machherndl-Spandl, Sigrid
%A Basara, Nadezda
%A Leis, Claudia
%A Schrade, Anika
%A Kapp-Schwoerer, Silke
%A Cocciardi, Sibylle
%A Bullinger, Lars
%A Thol, Felicitas
%A Heuser, Michael
%A Paschka, Peter
%A Gaidzik, Verena I
%A Saadati, Maral
%A Benner, Axel
%A Schlenk, Richard F
%A Döhner, Konstanze
%A Ganser, Arnold
%T Intensive chemotherapy with or without gemtuzumab ozogamicin in patients with NPM1-mutated acute myeloid leukaemia (AMLSG 09-09): a randomised, open-label, multicentre, phase 3 trial.
%J The lancet  / Haematology
%V 10
%N 7
%@ 2352-3026
%C London [u.a.]
%I Elsevier
%M DKFZ-2023-00992
%P e495-e509
%D 2023
%Z 2023 Jul;10(7):e495-e509
%X Acute myeloid leukaemia with mutated NPM1 is associated with high CD33 expression and intermediate-risk cytogenetics. The aim of this study was to evaluate intensive chemotherapy with or without the anti-CD33 antibody-drug conjugate gemtuzumab ozogamicin in participants with newly diagnosed, NPM1-mutated acute myeloid leukaemia.This open-label, phase 3 trial was conducted at 56 hospitals in Germany and Austria. Eligible participants were 18 years or older and had newly diagnosed NPM1-mutated acute myeloid leukaemia and an Eastern Cooperative Oncology Group performance status of 0-2. Participants were randomly assigned, using age as a stratification factor (18-60 years vs >60 years), 1:1 to the two treatment groups using allocation concealment; there was no masking of participants and investigators to treatment groups. Participants received two cycles of induction therapy (idarubicin, cytarabine, and etoposide) plus all-trans retinoic acid (ATRA) followed by three consolidation cycles of high-dose cytarabine (or an intermediate dose for those older than 60 years) and ATRA, without or with gemtuzumab ozogamicin (3 mg/m2 administered intravenously on day 1 of induction cycles 1 and 2, and consolidation cycle 1). The primary endpoints were short-term event-free survival and overall survival in the intention-to-treat population (overall survival was added as a co-primary endpoint after amendment four of the protocol on Oct 13, 2013). The secondary endpoints were event-free survival with long-term follow-up, rates of complete remission, complete remission with partial haematological recovery (CRh), and complete remission with incomplete haematological recovery (CRi), cumulative incidences of relapse and death, and number of days in hospital. This trial is registered with ClinicalTrials.gov (NCT00893399) and has been completed.Between May 12, 2010, and Sept 1, 2017, 600 participants were enrolled, of which 588 (315 women and 273 men) were randomly assigned (296 to the standard group and 292 to the gemtuzumab ozogamicin group). No difference was found in short-term event-free survival (short-term event-free survival at 6-month follow-up, 53
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:37187198
%R 10.1016/S2352-3026(23)00089-3
%U https://inrepo02.dkfz.de/record/276086