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Multicenter Study
. 2025 Sep;39(9):2174-2180.
doi: 10.1038/s41375-025-02700-9. Epub 2025 Jul 21.

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

Julian Ronnacker #  1   2 , Philippe J Muller #  2   3   4 , Jan-Henrik Mikesch  1   2 , Sven Zukunft  5 , Barbora Weinbergerová  6 , Jiří Šrámek  7 , Jan Valka  8 , Jan Novak  9 , Pavel Zak  10 , Tomas Szotkowski  11 , Zdenek Koristek  12 , Carolin Krekeler  1   2 , Julia M Unglaub  13 , Tim Sauer  13 , Leo Ruhnke  5 , Sabrina Kraus  14 , Judith Schaffrath  15 , Lutz P Müller  15 , Sabrina Kaes  16 , Dirk Niemann  16 , Lars Fransecky  17 , Patrick P Hess  18 , Martina Crysandt  18 , Edgar Jost  18 , Joana Millo  19 , Johannes Gaertner  20 , Roland Repp  21 , Madlen Jentzsch  22 , Lea Hoppe  23 , Stefan Klein  23 , Franziska Modemann  24 , Nina Michalowski  25 , Klaudia Fischbach  26 , Wolfgang Blau  26 , Marion Ruhs  27 , Markus Ritter  27 , Julian Lohmeyer  28 , Björn Steffen  28 , Sarah Hauser  29 , Martin Kaufmann  28 , Stefan W Krause  30 , Ricarda Knabe  31 , Karsten Spiekermann  31 , Hubert Serve  28 , Uwe Platzbecker  22 , Claudia D Baldus  17 , Carsten Müller-Tidow  13 , Georg Lenz  1   2 , Hans Christian Reinhardt  2   3 , Jirí Mayer  6 , Martin Bornhäuser  5 , Christoph Röllig  5 , Christoph Schliemann #  32   33 , Maher Hanoun #  34   35
Affiliations
Multicenter Study

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

Julian Ronnacker et al. Leukemia. 2025 Sep.

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.

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Conflict of interest statement

Competing interests: TS received honorarium, financial support, and congress participation from Pfizer and served as a consultant for Pfizer. FM received support for meeting attendance from Servier, AbbVie, Incyte, Gilead, Jazz Pharmaceuticals, Novartis, Teva, Pfizer, and Amgen; received support for medical writing from Servier, and Springer Verlag; received research grants from Apis Technologies, and Daiichi Sankyo; received speaker honoraria from Servier, Jazz Pharmaceuticals, Daiichi Sankyo, and AbbVie; participated in advisory boards for Otsuka Pharma, Servier, Pfizer, and Daiichi Sankyo. SK received honorarium as a speaker from Eickeler and travel support from AbbVie, Alexion, and Jazz. KS received speaker honoraria and/or travel grants from and/or has been a consultant to: AbbVie, Alexa, Bristol Myers Squibb, Jazz Pharmaceuticals, Leo, Pfizer, Sanofi, Servier, Sobi, Stemline, Takeda, MSD, Gilead. GL received research grants not related to this manuscript from AGIOS, AQUINOX, AstraZeneca, Bayer, Celgene, Gilead, Janssen, MorphoSys, Novartis, F. Hoffmann-La Roche Ltd, and Verastem. GL received honoraria not related to this study from ADC Therapeutics, AbbVie, Amgen, AstraZeneca, Bayer, BeiGene, BMS, Celgene, Constellation, Genase, Genmab, Gilead, Hexal/Sandoz, Immagene, Incyte, Janssen, Karyopharm, Lilly, Miltenyi, MorphoSys, MSD, NanoString, Novartis, PentixaPharm, Pierre Fabre, F. Hoffmann-La Roche Ltd, and Sobi. HCR received consulting and lecture fees from AbbVie, Roche, KinSea, Vitis, Cerus, Lilly, Novartis, Takeda, AstraZeneca, Vertex, and Merck. HCR received research funding from AstraZeneca and Gilead Pharmaceuticals. HCR is a co-founder of CDL Therapeutics GmbH. MBo has no conflict of interest regarding this work. Outside this study, he has received lecture fees and served on advisory boards for Jazz, MSD, and ActiTrexx. CR has received honoraria from AbbVie, Astellas, Bristol-Meyer-Squibb, Daiichi Sankyo, Jazz, Janssen, Novartis, Otsuka, Pfizer, Servier, and institutional research funding from AbbVie, Astellas, Novartis, Pfizer. CS received honoraria/has served on Advisory Boards for AbbVie, Astellas, AstraZeneca, BMS, Daiichi Sankyo, Laboratories Delbert, Jazz Pharmaceuticals, Novartis, Otsuka, Pfizer, Roche. CS received institutional research support from Jazz Pharmaceuticals and congress/travel grants from AbbVie, BMS, Jazz Pharmaceuticals, and Pfizer. Ethics approval and consent to participate: All procedures were performed in accordance with the relevant guidelines and regulations. The bio-registry of the SAL (NCT03188874), which encompasses 59 centers specialized in the treatment of hematologic neoplasms across Germany and the Czech Republic, is approved by the ethics committee of the Technical University Dresden (EK 98032010). The registry at the University Hospital Hamburg-Eppendorf is approved by the ethics committee of the Medical Association Hamburg (PV3469). Written informed consent was obtained from all participants.

Figures

Fig. 1
Fig. 1. Time-to-event outcomes in GO- vs. non-GO-treated patients.
The addition of GO was associated with significantly improved OS (A) and EFS (B). CIR was numerically lower in GO-treated patients compared to those who did not receive GO (C), although the difference did not reach statistical significance. GO gemtuzumab ozogamicin, HR hazard ratio, CI confidence interval.
Fig. 2
Fig. 2. Subgroup analysis of the effect of GO treatment on overall survival.
No heterogeneity in treatment effect was detected across subgroups, as assessed by tests for interaction. Missing values for ECOG, WBC, karyotype aberrations, and co-mutations were not imputed. CBF core-binding factor, ECOG Eastern Cooperative Oncology Group performance score, GO gemtuzumab ozogamicin, HR hazard ratio, CI confidence interval, WBC white blood count.
See this image and copyright information in PMC

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