吉妥珠单抗奥佐米星用于核心结合因子急性髓系白血病一线治疗：一项回顾性多中心分析的见解

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

作者信息

Ronnacker Julian, Muller Philippe J, Mikesch Jan-Henrik, Zukunft Sven, Weinbergerová Barbora, Šrámek Jiří, Valka Jan, Novak Jan, Zak Pavel, Szotkowski Tomas, Koristek Zdenek, Krekeler Carolin, Unglaub Julia M, Sauer Tim, Ruhnke Leo, Kraus Sabrina, Schaffrath Judith, Müller 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 Björn, Hauser Sarah, Kaufmann Martin, Krause Stefan W, Knabe Ricarda, Spiekermann Karsten, Serve Hubert, Platzbecker Uwe, Baldus Claudia D, Müller-Tidow Carsten, Lenz Georg, Reinhardt Hans Christian, Mayer Jirí, Bornhäuser Martin, Röllig Christoph, Schliemann Christoph, Hanoun Maher

机构信息

Department of Medicine A, University Hospital Münster, Münster, Germany.

West German Cancer Center (WTZ), Essen-Münster, Germany.

出版信息

Leukemia. 2025 Jul 21. doi: 10.1038/s41375-025-02700-9.

DOI:10.1038/s41375-025-02700-9

PMID:40691504

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.

摘要

在强化化疗（IC）中添加吉妥珠单抗奥唑米星（GO）已成为治疗核心结合因子急性髓系白血病（CBF-AML）患者的主要手段。然而，GO在这一特定亚组中疗效的证据主要基于十多年前进行的不同试验的荟萃分析数据。在这项基于登记处的研究中，我们评估了在来自SAL、AMLCG和CELL合作研究组的265例CBF-AML患者中添加GO至IC的影响。接受GO治疗的患者2年总生存率为90%，而未接受GO治疗的患者为80%（风险比[HR]0.45，95%置信区间[CI]0.21 - 0.95，P = 0.036），2年无事件生存率为51%，而未接受GO治疗者为36%（HR 0.69，95%CI 0.48 - 0.99，P = 0.046）。虽然接受GO与未接受GO治疗的患者完全缓解率相当（89%对90%，P = 0.81），但更多接受GO治疗的患者实现了微小残留病阴性缓解（77%对49%，P < 0.001），导致复发的累积发生率在数值上降低（HR 0.67，95%CI 0.43 - ！1.02，P = 0.06）。尽管血小板恢复延迟，但接受GO治疗的患者中重度毒性并未增加。这些发现支持将GO纳入适合接受IC治疗的CBF-AML患者的治疗方案中。