肿瘤细胞治疗研究
英文原题：Real-World Experience with Approved CAR T-Cell Therapies Ciltacabtagene Autoleucel and Idecabtagene Vicleucel in 1272 Relapsed/Refractory Multiple Myeloma Patients.
结果：共识别出697例接受cilta-cel治疗的患者和575例接受ide-cel治疗的患者。
背景：Ciltacabtagene autoleucel（cilta-cel）和idecabtagene vicleucel（ide-cel）已改变了复发/难治性多发性骨髓瘤（RRMM）的治疗格局。鉴于其近期获批及可及性有限（主要由于物流问题），真实世界数据仍然稀缺。方法：使用TriNetX数据库进行了一项回顾性研究，纳入接受cilta-cel或ide-cel治疗的成人RRMM患者。评估的临床结局包括总生存期（OS）、无进展生存期（PFS）以及安全性特征。结果：共识别出697例接受cilta-cel治疗和575例接受ide-cel治疗的患者。中位年龄分别为65岁和67岁，约16%为Black/African American。12个月OS在cilta-cel组为89.6%，在ide-cel组为86.0%。在一项描述性亚组分析中，肾功能损害（eGFR < 60 mL/min/1.73 m 2）似乎与两个队列中显著更差的OS相关（cilta-cel：HR = 3.66，p < 0.001；ide-cel：HR = 1.73，p = 0.003）。相反，既往抗CD38暴露似乎未影响两个治疗组中任一组的生存。任何级别CRS发生于45.9%（cilta-cel）和41.8%（ide-cel），而任何级别ICANS分别见于15.4%和11.8%。严重（3级）ICANS在两个队列中仍然罕见（<3%）。血液学毒性普遍存在，3级中性粒细胞减少发生于76.0%（cilta-cel）和68.0%（ide-cel）。值得注意的是，任何级别感染（28.5-40.1%）和低丙种球蛋白血症（41.1-43.1%）较为常见，突显了显著的长期免疫抑制负担。结论：在这些真实世界队列中，两种已获批的CAR T细胞疗法均显示出良好的生存结局。尽管严重血液学和免疫相关毒性的发生率较高，但这些发现与已发表的临床试验数据一致，并且这些药物的临床实用性似乎超过了其不良安全性特征。
Background: Ciltacabtagene autoleucel (cilta-cel) and idecabtagene vicleucel (ide-cel) have transformed the treatment landscape of relapsed/refractory multiple myeloma (RRMM). Given their recent regulatory approval and limited availability, mainly due to logistical issues, real-world data remain scarce. Methods: A retrospective study was conducted using the TriNetX database, identifying adult patients with RRMM treated with either cilta-cel or ide-cel. The clinical outcomes evaluated included overall survival (OS), progression-free survival (PFS), as well as the safety profile. Results: A total of 697 patients treated with cilta-cel and 575 with ide-cel were identified. The median age was 65 and 67 years, with ~16% being Black/African American. The 12-month OS was 89.6% for cilta-cel and 86.0% for ide-cel. In a descriptive subgroup analysis, renal impairment (eGFR < 60 mL/min/1.73 m 2 ) seemed to be associated with significantly inferior OS in both cohorts (HR = 3.66, p < 0.001 for cilta-cel; HR = 1.73, p = 0.003 for ide-cel). Conversely, prior anti-CD38 exposure did not seem to impact survival in any of the two treatment groups. Any-grade CRS occurred in 45.9% (cilta-cel) and 41.8% (ide-cel), while any-grade ICANS was observed in 15.4% and 11.8%, respectively. Severe (grade 3) ICANS remained rare (<3%) in both cohorts. Hematologic toxicity was prevalent, with grade 3 neutropenia occurring in 76.0% (cilta-cel) and 68.0% (ide-cel). Notably, any-grade infections (28.5-40.1%) and hypogammaglobulinemia (41.1-43.1%) were frequent, highlighting a significant long-term immunosuppressive burden. Conclusions: In these real-world cohorts, both approved CAR T-cell therapies demonstrated favorable survival outcomes. While the incidence of severe hematologic and immune-related toxicities was high, these findings are compatible with published data from clinical trials and it seems that the clinical utility of these drugs overcomes the adverse safety profile.
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