Gemtuzumab ozogamicin in first-line treatment of CBF-AML: insights from a retrospective multi-center analysis

Ronnacker J, Muller PJ, Mikesch JH, Zukunft S, Weinbergerová B, Šrámek J, Valka J, Novak J, Zak P, Szotkowski T, Koristek Z, Krekeler C, Unglaub JM, Sauer T, Ruhnke L, Kraus S, Schaffrath J, Müller LP, Kaes S, Niemann D, Fransecky L, Hess PP, Crysandt M, Jost E, Millo J, Gaertner J, Repp R, Jentzsch M, Hoppe L, Klein S, Modemann F, Michalowski N, Fischbach K, Blau W, Ruhs M, Ritter M, Lohmeyer J, Steffen B, Hauser S, Kaufmann M, Krause S, Knabe R, Spiekermann K, Serve H, Platzbecker U, Baldus CD, Müller-Tidow C, Lenz G, Reinhardt HC, Mayer J, Bornhäuser M, Röllig C, Schliemann C, Hanoun M (2025)

Publication Type: Journal article

Publication year: 2025

Journal

DOI: 10.1038/s41375-025-02700-9

Abstract

The addition of gemtuzumab ozogamicin (GO) to intensive chemotherapy (IC) has become a mainstay in treating patients with core binding factor acute myeloid leukemia (CBF-AML). However, evidence for the efficacy of GO in this particular subgroup is primarily based on meta-analytic data from different trials conducted more than a decade ago. In this registry-based study, we evaluated the impact of adding GO to IC in 265 CBF-AML patients from the SAL, AMLCG, and CELL cooperative study groups. Patients receiving GO had a 2-year overall survival of 90% compared with 80% in those without GO (hazard ratio [HR] 0.45, 95% confidence interval [CI] 0.21–0.95, P = 0.036) and a 2-year event-free survival of 51% versus 36% (HR 0.69, 95% CI 0.48–0.99, P = 0.046). While complete remission rates in GO vs. non-GO patients were comparable (89% vs. 90%, P = 0.81), more GO patients achieved measurable residual disease-negative remission (77% vs. 49%, P < 0.001), resulting in numerically reduced cumulative incidence of relapse (HR 0.67, 95% CI 0.43–1.02, P = 0.06). Despite delayed platelet recovery, high-grade toxicities were not increased in GO-treated patients. These findings support the integration of GO into treatment protocols for IC-eligible patients with CBF-AML.

Authors with CRIS profile

Involved external institutions

Universitätsklinikum Münster Germany (DE) Universitätsklinikum Essen Germany (DE) Universitätsklinikum Carl Gustav Carus Dresden Germany (DE) University Hospital Brno Czech Republic (CZ) Fakultní Nemocnice Plzeň Czech Republic (CZ) Fakultní nemocnice Královské Vinohrady Czech Republic (CZ) University Hospital Hradec Králové / Fakultní Nemocnice Hradec Králové Czech Republic (CZ) Palacky University Olomouc / Univerzita Palackého v Olomouci Czech Republic (CZ) University Hospital Ostrava / Fakultní Nemocnice Ostrava Czech Republic (CZ) Universitätsklinikum Heidelberg Germany (DE) Universitätsklinikum Würzburg Germany (DE) Universitätsklinikum Halle (Saale) Germany (DE) Universitätsklinikum Schleswig-Holstein (UKSH) Germany (DE) Universitätsklinikum Aachen (UKA) Germany (DE) Rems-Murr-Kliniken Germany (DE) Klinikum Nürnberg Germany (DE) Städtisches Krankenhaus Kiel Germany (DE) Horst-Schmidt-Kliniken Germany (DE) Universitätsklinikum Frankfurt am Main (KGU) Germany (DE) Universitätsklinikum Leipzig Germany (DE) Klinikverbund Südwest (Sindelfingen-Böblingen, Calw, Nagold, Leonberg, Herrenberg) Germany (DE) Robert-Bosch-Krankenhaus Germany (DE) Klinikum der Universität München Germany (DE) Universitätsklinikum Mannheim Germany (DE) Universitätsklinikum Hamburg-Eppendorf (UKE) Germany (DE)

How to cite

APA:

Ronnacker, J., Muller, P.J., Mikesch, J.H., Zukunft, S., Weinbergerová, B., Šrámek, J.,... Hanoun, M. (2025). Gemtuzumab ozogamicin in first-line treatment of CBF-AML: insights from a retrospective multi-center analysis. Leukemia. https://doi.org/10.1038/s41375-025-02700-9

MLA:

Ronnacker, Julian, et al. "Gemtuzumab ozogamicin in first-line treatment of CBF-AML: insights from a retrospective multi-center analysis." Leukemia (2025).

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