Publication details

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

Authors	RONNACKER Julian MULLER Philippe J MIKESCH Jan-Henrik ZUKUNFT Sven WEINBERGEROVÁ Barbora SRAMEK Jiri VALKA Jan NOVAK Jan ZAK Pavel SZOTKOWSKI Tomas KORISTEK Zdenek KREKELER Carolin UNGLAUB Julia M SAUER Tim RUHNKE Leo KRAUS Sabrina SCHAFFRATH Judith MUELLER Lutz P KAES Sabrina NIEMANN Dirk FRANSECKY Lars HESS Patrick P CRYSANDT Martina JOST Edgar MILLO Joana GAERTNER Johannes REPP Roland JENTZSCH Madlen HOPPE Lea KLEIN Stefan MODEMANN Franziska MICHALOWSKI Nina FISCHBACH Klaudia BLAU Wolfgang RUHS Marion RITTER Markus LOHMEYER Julian STEFFEN Bjoern HAUSER Sarah KAUFMANN Martin KRAUSE Stefan W KNABE Ricarda SPIEKERMANN Karsten SERVE Hubert PLATZBECKER Uwe BALDUS Claudia D MUELLER-TIDOW Carsten LENZ Georg REINHARDT Hans Christian MAYER Jiří BORNHAEUSER Martin ROELLIG Christoph SCHLIEMANN Christoph HANOUN Maher
Year of publication	2025
Type	Article in Periodical
Magazine / Source	Leukemia
MU Faculty or unit	Faculty of Medicine
Citation
web	https://www.nature.com/articles/s41375-025-02700-9
Doi	https://doi.org/10.1038/s41375-025-02700-9
Description	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.