CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
比较 ciltacabtagene autoleucel 在 CARTITUDE-1 研究与 Flatiron Health 多发性骨髓瘤队列注册研究中医生选择疗法对复发/难治性多发性骨髓瘤患者治疗的疗效
英文原题：Comparative effectiveness of ciltacabtagene autoleucel in CARTITUDE-1 versus physician's choice of therapy in the Flatiron Health multiple myeloma cohort registry for the treatment of patients with relapsed or refractory multiple myeloma.
查看英文原题
Comparative effectiveness of ciltacabtagene autoleucel in CARTITUDE-1 versus physician's choice of therapy in the Flatiron Health multiple myeloma cohort registry for the treatment of patients with relapsed or refractory multiple myeloma.
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研究概要
Cilta-cel 在所有结局上均显示出显著优于 PCT 的有效性，突显其作为三药暴露 RRMM 患者有效疗法的潜力。
研究思路结论见上方概要
背景
西达基奥仑赛是一种新型CAR-T 细胞疗法，正在CARTITUDE-1试验中评估，该试验针对既往接受过免疫调节药物、蛋白酶体抑制剂和抗CD38单克隆抗体治疗的复发/难治性多发性骨髓瘤患者。鉴于CARTITUDE-1缺乏对照组，本研究利用Flatiron Health多发性骨髓瘤队列登记处的外部真实世界对照臂，评估了西达基奥仑赛与医生选择治疗的比较有效性。
方法
鉴于CARTITUDE-1和Flatiron中PCT提供了cilta-cel的个体患者数据，采用逆概率治疗加权来调整具有预后意义的基线协变量不平衡：难治状态、细胞遗传学特征、国际分期系统分期、末次治疗方案至进展时间、既往治疗线数、自诊断以来的年数以及年龄。比较了无进展生存期（PFS）、至下次治疗时间（TTNT）和总生存期（OS）的有效性。并进行了一系列敏感性分析。
结果
经过倾向评分加权后，两个队列的基线特征相似。与PCT相比，接受cilta-cel治疗的患者PFS（HR：0.18 [95% CI：0.12，0.27；p < 0.0001]）、TTNT（HR：0.15 [95% CI：0.09，0.22；p < 0.0001]）和OS（HR：0.25 [95% CI：0.13，0.46；p < 0.0001]）均有所改善。cilta-cel的治疗获益在所有敏感性分析中均稳健且一致。
展开英文摘要原文
METHODS
Given the availability of individual patient data for cilta-cel from CARTITUDE-1 and PCT in Flatiron, inverse probability of treatment weighting was used to adjust for unbalanced baseline covariates of prognostic significance: refractory status, cytogenetic profile, International Staging System stage, time to progression on last regimen, number of prior lines of therapy, years since diagnosis, and age. Comparative effectiveness was estimated for progression-free survival (PFS), time to next treatment (TTNT), and overall survival (OS). A range of sensitivity analyses were conducted.
RESULTS
Baseline characteristics were similar between the two cohorts after propensity score weighting. Patients with cilta-cel had improved PFS (HR: 0.18 [95% CI: 0.12, 0.27; p < 0.0001]), TTNT (HR: 0.15 [95% CI: 0.09, 0.22; p < 0.0001]), and OS (HR: 0.25 [95% CI: 0.13, 0.46; p < 0.0001]) versus PCT. Cilta-cel treatment benefit was robust and consistent across all sensitivity analyses.
CONCLUSION
Cilta-cel demonstrated significantly superior effectiveness over PCT for all outcomes, highlighting its potential as an effective therapy in patients with triple-class exposed RRMM.
论文信息
- 作者
- Martin T、Krishnan A、Yong K、Weisel K、Mehra M、Nair S、Qi K、Londhe A
- 第一作者单位
- UCSF Helen Diller Family Comprehensive Cancer Center San Francisco California USA.United States
- 通讯作者单位
- Mount Sinai Medical Center New York New York USA.United States
- 期刊
- EJHaem2022 Feb
- 原文标识
- PubMed 35846215 · DOI 10.1002/jha2.312
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