A phase 3 study of intensive chemotherapy with or without dasatinib in core-binding factor acute myeloid leukemia

H Hartmut Döhner (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) D Daniela Späth (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) M Maral Saadati W Walter Fiedler K Katharina Götze (4Department of Medicine III, Hematology and Medical Oncology, Technical University of Munich, Munich, Germany) E Elisabeth Koller (5Department of Internal Medicine III, Hanuschkrankenhaus Wien, Vienna, Austria) J Jörg Westermann (6Department of Hematology, Oncology, and Cancer Immunology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany) W Wichard Vogel (7Department of Hematology and Oncology, Eberhard-Karls University, Tübingen, Germany) M Michael Heuser M Michael Lübbert (10Department of Medicine I, Medical Center–University of Freiburg, Faculty of Medicine, University of Freiburg, Germany) H Hans-Joachim Tischler (11Department of Hematology and Oncology, University Hospital of Minden, Minden, Germany) U Ulrich Germing L Lino L. Teichmann L Lars Fransecky (14Department of Internal Medicine II, University Hospital Schleswig Holstein, Campus Kiel, Kiel, Germany) A Albert Wölfler (15Division of Hematology, Department of Internal Medicine, Medical University of Graz, Graz, Austria) D David Nachbaur (16Internal Medicine V, Hematology and Oncology, Medical University of Innsbruck, Innsbruck, Austria) B Bernd Hertenstein (17Department of Hematology and Oncology, Klinikum Bremen-Mitte, Bremen, Germany) R Roland Schroers (18Medical Clinic II-Hematology and Oncology, Ruhr-University Bochum, Bochum, Germany) U Uwe Martens (19Department of Hematology and Oncology, SLK-Clinics Heilbronn GmbH, Heilbronn, Germany) S Stephanie von Harsdorf (20Klinik für Hämatologie, Onkologie & Stammzelltransplantation, Evangelische Kliniken Essen-Mitte, Essen, Germany) M Markus Radsak (1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany) G Gregor Aschauer (22Internal Medicine I, Medical Oncology, Hematology and Gastroenterology, Barmherzige Schwestern Hospital, Linz, Austria) S Stefanie Weißhaar (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) A Andrea Corbacioglu (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) A Anika Schrade (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) V Verena I. Gaidzik (1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany) F Felicitas Thol P Peter Paschka (23Klinikum der Stadt Ludwigshafen am Rhein, Medizinische Klinik A, Ludwigshafen am Rhein, Germany) L Lars Bullinger A Axel Benner K Konstanze Döhner (12University Hospital of Ulm, Ulm, Germany) A Arnold Ganser (8Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany)

Abstract

Abstract Core-binding factor acute myeloid leukemia (CBF-AML) is associated with KIT mutations and deregulated expression of KIT. We report results from the randomized, open-label, phase 3 trial of intensive chemotherapy with or without the multikinase inhibitor dasatinib in adult patients with CBF-AML. Patients received “3+7” induction therapy, followed by 4 cycles of high-dose cytarabine; in the investigational arm, patients received dasatinib 100 mg daily on days 8 to 21 in induction, and on days 6 to 28 in consolidation cycles, followed by 12-month single-agent dasatinib 100 mg daily. Primary end point was event-free survival (EFS). Secondary end points included overall survival, relapse-free survival, and cumulative incidence of relapse. A total of 202 patients were randomly assigned to the standard arm (n = 102) and to the dasatinib arm (n = 100). Median age was 49 years (range, 18-77); 94 patients had t(8;21), 108 had inv(16)/t(16;16); and 58 (28.7%) patients had a KIT comutation. There was no statistically significant difference in EFS (hazard ratio, 0.92; 95% confidence interval, 0.63-1.33; P = .66) or secondary end points between treatment arms. There was also no significant difference in EFS in subgroup analyses according to age, CBF-AML type, and KIT mutation status. The incidence of serious adverse events was higher in the investigational arm (64%) than in the standard arm (36%). In patients with CBF-AML, the addition of dasatinib to intensive chemotherapy failed to improve survival outcomes. The addition of dasatinib was associated with an increase in toxicity. This trial was registered at www.ClinicalTrials.gov as NCT02013648.

Article Details

Journal Blood
Volume / Issue Vol. 147, Issue 15
Published April 09, 2026
Pages 1735-1748
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (32)

H

Hartmut Döhner

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

D

Daniela Späth

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

M

Maral Saadati

W

Walter Fiedler

K

Katharina Götze

4Department of Medicine III, Hematology and Medical Oncology, Technical University of Munich, Munich, Germany

E

Elisabeth Koller

5Department of Internal Medicine III, Hanuschkrankenhaus Wien, Vienna, Austria

J

Jörg Westermann

6Department of Hematology, Oncology, and Cancer Immunology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany

W

Wichard Vogel

7Department of Hematology and Oncology, Eberhard-Karls University, Tübingen, Germany

M

Michael Heuser

M

Michael Lübbert

10Department of Medicine I, Medical Center–University of Freiburg, Faculty of Medicine, University of Freiburg, Germany

H

Hans-Joachim Tischler

11Department of Hematology and Oncology, University Hospital of Minden, Minden, Germany

U

Ulrich Germing

L

Lino L. Teichmann

L

Lars Fransecky

14Department of Internal Medicine II, University Hospital Schleswig Holstein, Campus Kiel, Kiel, Germany

A

Albert Wölfler

15Division of Hematology, Department of Internal Medicine, Medical University of Graz, Graz, Austria

D

David Nachbaur

16Internal Medicine V, Hematology and Oncology, Medical University of Innsbruck, Innsbruck, Austria

B

Bernd Hertenstein

17Department of Hematology and Oncology, Klinikum Bremen-Mitte, Bremen, Germany

R

Roland Schroers

18Medical Clinic II-Hematology and Oncology, Ruhr-University Bochum, Bochum, Germany

U

Uwe Martens

19Department of Hematology and Oncology, SLK-Clinics Heilbronn GmbH, Heilbronn, Germany

S

Stephanie von Harsdorf

20Klinik für Hämatologie, Onkologie & Stammzelltransplantation, Evangelische Kliniken Essen-Mitte, Essen, Germany

M

Markus Radsak

1Department of Hematology and Oncology, Medical Center of the Johannes Gutenberg University Mainz, Mainz, Germany

G

Gregor Aschauer

22Internal Medicine I, Medical Oncology, Hematology and Gastroenterology, Barmherzige Schwestern Hospital, Linz, Austria

S

Stefanie Weißhaar

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

A

Andrea Corbacioglu

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

A

Anika Schrade

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

V

Verena I. Gaidzik

1Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany

F

Felicitas Thol

P

Peter Paschka

23Klinikum der Stadt Ludwigshafen am Rhein, Medizinische Klinik A, Ludwigshafen am Rhein, Germany

L

Lars Bullinger

A

Axel Benner

K

Konstanze Döhner

12University Hospital of Ulm, Ulm, Germany

A

Arnold Ganser

8Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany