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.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:A Cost-Effectiveness Analysis of Axicabtagene Ciloleucel versus Tisagenlecleucel in the Treatment of Diffuse Large B-cell Lymphoma Based on a Real-World French Registry.
A Cost-Effectiveness Analysis of Axicabtagene Ciloleucel versus Tisagenlecleucel in the Treatment of Diffuse Large B-cell Lymphoma Based on a Real-World French Registry.
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基于欧洲采用的明确支付意愿阈值,在 3L+ r/r DLBCL 中,与 tisa-cel 相比,axi-cel 预计在真实世界临床环境中是医疗资源的一种具有成本效益的使用方式。
Axicabtagene ciloleucel(axi-cel)和tisagenlecleucel(tisa-cel)是CAR-T 细胞疗法,分别在ZUMA-1和JULIET试验中用于评估三线及以后(3L+)复发/难治性(r/r)弥漫性大B细胞淋巴瘤(DLBCL)。截至2021年10月,DESCAR-T 注册研究纳入了729例接受axi-cel或tisa-cel治疗的法国3L+ r/r DLBCL患者。利用这些数据,采用倾向性评分匹配对axi-cel与tisa-cel进行校正比较。与tisa-cel相比,axi-cel与总生存期(OS)和无进展生存期(PFS)的统计学显著改善相关,且3级免疫效应细胞相关神经毒性综合征(ICANS)的发生率显著更高。3级细胞因子释放综合征(CRS)无显著差异。本分析利用DESCAR-T 注册研究的倾向性评分匹配数据,评估了axi-cel与tisa-cel在治疗3L+ r/r DLBCL中的成本效益。
使用分区生存模型将成本和QALYs外推至终生。PFS和OS的生存曲线基于独立的混合治愈模型,拟合自倾向评分匹配的DESCAR-T 人群的数字化Kaplan-Meier数据。DESCAR-T 中axi-cel和tisa-cel各自的平均ICU住院时间用于估算不良事件成本。选定的参数生存分布基于临床专家验证。效用值来源于ZUMA-1,成本来自法国登记处和已发表来源。axi-cel和tisa-cel均使用标价。成本和结局按每年2.5%的贴现率进行贴现。
与tisa-cel相比,Axi-cel的增量成本效果比为每QALY 15,520。
A partitioned survival model was used to extrapolate costs and quality-adjusted life years (QALYs) over a lifetime. Survival curves for PFS and OS were based on independent mixture cure models fitted to digitized Kaplan-Meier data for the propensity score-matched DESCAR-T populations. Average duration of intensive care unit stays for each of axi-cel and tisa-cel in DESCAR-T were used to inform adverse event costs. Selected parametric survival distributions were based on clinical expert validation. Utility values were derived from ZUMA-1, and costs were obtained from French registries and published sources. List prices were used for both axi-cel and tisa-cel. Costs and outcomes were discounted at an annual rate of 2.5%.
Axi-cel is associated with an incremental cost-effectiveness ratio of 15,520 per QALY compared with tisa-cel.
Based on explicit willingness-to-pay thresholds applied in Europe, axi-cel is expected to be a cost-effective use of healthcare resources in real-world clinical settings compared with tisa-cel in 3L+ r/r DLBCL.
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