Department of Immuno-Hematology, Hôpital Saint Louis, APHP/Université Paris Cité, Paris, France.
Inserm U976 HIPI, Team 5: Lymphocyte Development and Lymphoid Disorders, Hôpital Saint Louis, Paris, France.
Br J Haematol. 2024 Sep;205(3):990-998. doi: 10.1111/bjh.19505. Epub 2024 May 15.
Idecabtagene vicleucel (ide-cel), a chimeric antigen receptor T-cell therapy targeting B-cell maturation antigen (BCMA), received early access program (EAP) authorization in France in April 2021 for relapsed/refractory multiple myeloma (RRMM). We conducted a real-world registry-based multicentre observational study in 11 French hospitals to evaluate ide-cel outcomes. Data from 176 RRMM patients who underwent apheresis between June 2021 and November 2022 were collected from the French national DESCAR-T registry. Of these, 159 patients (90%) received ide-cel. Cytokine release syndrome occurred in 90% with 2% grade ≥3, and neurotoxicity occurred in 12% with 3% grade ≥3. Over the first 6 months, the best overall response and ≥complete response rates were 88% and 47% respectively. The median progression-free survival (PFS) from the ide-cel infusion was 12.5 months, the median overall survival (OS) was 20.8 months and the estimated OS rate at 12 months was 73.3%. Patients with extra-medullary disease (EMD) had impaired PFS (6.2 months vs. 14.8 months). On multivariable analysis, EMD and previous exposure to BCMA-targeted immunoconjugate or T-cell-redirecting GPRC5D bispecific antibody were associated with inferior PFS. Our study supports ide-cel's feasibility, safety and efficacy in real-life settings, emphasizing the importance of screening for EMD and considering prior treatments to optimize patient selection.
伊达赛珠单抗(ide-cel)是一种嵌合抗原受体 T 细胞疗法,针对 B 细胞成熟抗原(BCMA),于 2021 年 4 月在法国获得了早期准入计划(EAP)的批准,用于治疗复发/难治性多发性骨髓瘤(RRMM)。我们在法国 11 家医院开展了一项基于真实世界登记的多中心观察性研究,以评估 ide-cel 的疗效。该研究从法国国家 DESCAR-T 登记处收集了 2021 年 6 月至 2022 年 11 月期间接受过单采的 176 例 RRMM 患者的数据。其中,159 例(90%)患者接受了 ide-cel 治疗。发生细胞因子释放综合征的患者占 90%(2%为 3 级及以上),发生神经毒性的患者占 12%(3%为 3 级及以上)。在最初的 6 个月内,最佳总体缓解率和≥完全缓解率分别为 88%和 47%。从 ide-cel 输注开始,中位无进展生存期(PFS)为 12.5 个月,中位总生存期(OS)为 20.8 个月,12 个月时的估计 OS 率为 73.3%。有髓外疾病(EMD)的患者 PFS 受损(6.2 个月 vs. 14.8 个月)。多变量分析显示,EMD 和先前暴露于 BCMA 靶向免疫偶联物或 T 细胞重定向 GPRC5D 双特异性抗体与较差的 PFS 相关。本研究支持 ide-cel 在真实环境中的可行性、安全性和疗效,强调了筛查 EMD 和考虑先前治疗以优化患者选择的重要性。