中文摘要
靶向B细胞成熟抗原的嵌合抗原受体(CAR)T细胞已彻底改变了复发/难治性多发性骨髓瘤的治疗。然而,目前尚无idecabtagene vicleucel(ide-cel)与ciltacabtagene autoleucel(cilta-cel)的随机头对头比较,真实世界数据提示两者在疗效和毒性方面存在差异。
我们对2024年1月至2025年7月期间接受ide-cel或cilta-cel治疗的奥地利患者进行了一项全国性回顾性真实世界分析(RWA)。共纳入90例患者,基线特征基本均衡,且高危特征常见。94.4%的患者接受了桥接治疗,在淋巴细胞清除前获得了较高的缓解率。中位随访17.6个月后,两种CAR-T 产品之间未观察到无进展生存期(PFS)曲线的早期分离。多变量分析显示,cilta-cel有改善PFS的趋势,而真性髓外病变(EMD)以及T细胞采集前接受过双特异性抗体治疗与较差的PFS相关。尽管既往RWA已证实cilta-cel与ide-cel之间PFS存在早期分离,但本队列中未观察到这一模式。桥接治疗的高有效性可能促成了这些发现。
我们的结果支持进一步研究最佳桥接策略和治疗序贯,同时强调真性EMD患者持续存在的未满足需求。
展开英文摘要原文
B-cell maturation antigen-directed chimeric antigen receptor (CAR) T cells have revolutionized the treatment of relapsed/refractory multiple myeloma.
However, no randomized head-to-head comparison of idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel) is available, and real-world data suggest differences in efficacy and toxicity.
We performed a nationwide retrospective real-world analysis (RWA) of Austrian patients treated with ide-cel or cilta-cel between January 2024 and July 2025. Ninety patients were included, with largely balanced baseline characteristics and frequent high-risk features. Bridging therapy was administered to 94. 4% of patients, resulting in high response rates prior to lymphodepletion. After a median follow-up of 17. 6 months, no early separation of the progression-free survival (PFS) curves was observed between CAR-T products.
Multivariable analyses showed a trend towards improved PFS with cilta-cel, whereas true extra-medullary disease (EMD) and prior bispecific antibody treatment before T-cell apheresis were associated with inferior PFS. Although previous RWA have demonstrated an early divergence in PFS between cilta-cel and ide-cel, this pattern was not observed in our cohort. The high effectiveness of bridging therapy may have contributed to these findings.
Our results support further investigation of optimal bridging strategies and treatment sequencing while highlighting the persistent unmet need of patients with true EMD.
论文信息
- 作者
- Strassl I、Nikoloudis A、Ruesing LZ、Melchardt T、Leisch M、Schulz E、Heine A、Neumeister P
- 第一作者单位
- Department of Internal Medicine I: Hematology With Stem Cell Transplantation, Hemostaseology and Medical Oncology, Ordensklinikum Linz Elisabethinen, Linz, Austria.Austria
- 通讯作者单位
- Division Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.Austria
- 期刊
- British journal of haematology2026 Aug 26