Treatment sequence with gilteritinib and allogeneic haematopoietic stem cell transplantation in relapsed/refractory FMS‐like tyrosine kinase 3 ( <scp>FLT3</scp> )‐mutated acute myeloid leukaemia patients: A multicentre real‐world study

Patients with relapsed or refractory acute myeloid leukaemia (r/r AML) harbouring FMS-like tyrosine kinase 3 (FLT3) mutations generally have poor prognosis and limited treatment options. The second-generation FLT3 inhibitor gilteritinib provides better disease control compared to standard relapse therapies and was approved based on two randomized trials. We analysed its efficacy and safety in a real-world cohort of r/r AML patients undergoing gilteritinib treatment including allogeneic haematopoietic stem cell transplantation (alloHSCT). Data were collected from all consecutive r/r AML patients with FLT3-internal tandem duplication and/or FLT3-tyrosine kinase domain mutations receiving gilteritinib at 25 centres. Response and survival were analysed using descriptive and correlative statistics. A total of 156 patients were analysed, 55.1% with relapse after previous alloHSCT, and 46.2% with prior FLT3 inhibitor exposure. The overall complete remission/incomplete remission (CR/Cri) rate was 45.3%, with a median overall survival (OS) of 10.0 months. Patients receiving gilteritinib only had a 1-year OS of 34%, while those with consolidating alloHSCT had a 1-year OS of 76%-92%, depending on gilteritinib maintenance post-transplant. Resuming gilteritinib post-transplant led to an 85.7% CR/CRi rate. Our real-world data confirm that gilteritinib is an effective single-agent therapy for r/r AML with FLT3 mutations, with best outcomes when used as a bridge to transplant and post-transplant maintenance.

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Journal
British Journal of Haematology
Published
2026-08-18
DOI
https://doi.org/10.1111/bjh.70735
Primary Topic
Acute Myeloid Leukemia Research
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article
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article

Treatment sequence with gilteritinib and allogeneic haematopoietic stem cell transplantation in relapsed/refractory FMS‐like tyrosine kinase 3 ( FLT3 )‐mutated acute myeloid leukaemia patients: A multicentre real‐world study

Guido Kobbe, Oliver Kriege, Paul Jäger, Johannes Schetelig et al.
British Journal of Haematology
Acute Myeloid Leukemia Research
article

Treatment sequence with gilteritinib and allogeneic haematopoietic stem cell transplantation in relapsed/refractory FMS‐like tyrosine kinase 3 ( FLT3 )‐mutated acute myeloid leukaemia patients: A multicentre real‐world study

Guido Kobbe, Oliver Kriege, Paul Jäger, Johannes Schetelig, Christian Thiede, Sven Zukunft, Elena Stauffer, Hermann Einsele, Judith Schaffrath, Wolfgang Andreas Bethge, Christoph Röllig, Martin Bornhäuser, Udo Holtick, Klaus Hans Metzeler, S K Metzelder, Tobias A.W. Holderried, Claudia Dorothea Baldus, Julian Ronnacker, Inken Hilgendorf, Tim H. Brümmendorf, Friedrich Stölzel, Jennifer Kaivers, Desireé Kunadt, Edgar Jost, Lambros Kordelas, Normann Steiner, David Nachbaur, Mathias Hänel, Jan Frederic Weller, Lars R. Fransecky, Sabrina Kraus, Christoph Schliemann, William Krüger, Anna Doleschall, Uwe Platzbecker, Anne Brandenburg, Jochen J. Frietsch, Tobias Herold, Gesine Bug, Julia M. Unglaub, Tim Sauer, Khouloud Kouidri, Eva Wagner‐Drouet, Jan M. Middeke, Sabine Kayser, Markus Maulhardt, Laura Reinhardt, Andreas Burchert, Nicolaus Kröger, for the German Cooperative Transplant Study Group and Study Alliance Leukemia (SAL), Andreas Hochhaus, Johanna Tischer, Elisa Sala, Björn Steffen, Matthias Stelljes, Thomas Schroeder
article en

Abstract

Patients with relapsed or refractory acute myeloid leukaemia (r/r AML) harbouring FMS-like tyrosine kinase 3 (FLT3) mutations generally have poor prognosis and limited treatment options. The second-generation FLT3 inhibitor gilteritinib provides better disease control compared to standard relapse therapies and was approved based on two randomized trials. We analysed its efficacy and safety in a real-world cohort of r/r AML patients undergoing gilteritinib treatment including allogeneic haematopoietic stem cell transplantation (alloHSCT). Data were collected from all consecutive r/r AML patients with FLT3-internal tandem duplication and/or FLT3-tyrosine kinase domain mutations receiving gilteritinib at 25 centres. Response and survival were analysed using descriptive and correlative statistics. A total of 156 patients were analysed, 55.1% with relapse after previous alloHSCT, and 46.2% with prior FLT3 inhibitor exposure. The overall complete remission/incomplete remission (CR/Cri) rate was 45.3%, with a median overall survival (OS) of 10.0 months. Patients receiving gilteritinib only had a 1-year OS of 34%, while those with consolidating alloHSCT had a 1-year OS of 76%-92%, depending on gilteritinib maintenance post-transplant. Resuming gilteritinib post-transplant led to an 85.7% CR/CRi rate. Our real-world data confirm that gilteritinib is an effective single-agent therapy for r/r AML with FLT3 mutations, with best outcomes when used as a bridge to transplant and post-transplant maintenance.

British Journal of Haematology
Goethe University Frankfurt (DE), Innsbruck Medical University (AT), Universität Hamburg (DE), Philipps University of Marburg (DE), German Cancer Research Center (DE), Johannes Gutenberg University Mainz (DE), Medical University of Graz (AT), Heidelberg University (DE), Universitätsmedizin Greifswald (DE), University Hospital Bonn (DE), University Medical Center Freiburg (DE), German Red Cross (DE), University Hospital Heidelberg (DE), LMU Klinikum (DE), München Klinik Bogenhausen (DE), Düsseldorf University Hospital (DE), University Medical Center of the Johannes Gutenberg University Mainz (DE), Augenklinik Universitätsklinikum Würzburg (DE), University Hospital Münster (DE), University Medical Center Hamburg-Eppendorf (DE), National Center for Tumor Diseases (DE), University Hospital Schleswig-Holstein (DE), Universitätsmedizin Göttingen (DE), Essen University Hospital (DE), Universitätsklinikum Aachen (DE), University Children's Hospital Tübingen (DE), University Hospital Leipzig (DE), Jena University Hospital (DE), University Hospital Cologne (DE), University Hospital Ulm (DE), Klinikum Chemnitz (DE), Deutsche Knochenmarkspenderdatei (DE), Klinik und Poliklinik für Psychotherapie und Psychosomatik (DE), Nationales Centrum für Tumorerkrankungen Dresden (DE), Heinrich Heine University Düsseldorf (DE), Martin Luther University Halle-Wittenberg (DE), University of Tübingen (DE), Ludwig-Maximilians-Universität München (DE), University of Lübeck (DE)
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