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:Cost-Effectiveness Analysis of Second-Line Lisocabtagene Maraleucel in the Treatment of Refractory or Relapsed Large B-Cell Lymphoma.
Cost-Effectiveness Analysis of Second-Line Lisocabtagene Maraleucel in the Treatment of Refractory or Relapsed Large B-Cell Lymphoma.
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在法国,liso-cel 相比标准治疗(SOC)治疗 R/R LBCL 患者具有成本效益。
**目的:**利索卡布他基因马拉赛(liso-cel)等CAR-T 细胞疗法已改变二线治疗原发难治或早期(12个月内)复发大B细胞淋巴瘤(LBCL)的方式。
本研究评估liso-cel相较标准治疗(SOC)用于法国复发/难治性LBCL的成本效果。**方法:**研究建立三健康状态模型,在法国以20年时间范围比较liso-cel与SOC。疗效和安全性数据外推自TRANSFORM试验(NCT03575351)或DESCAR-T(法国CAR-T 治疗患者登记队列)。成本依据法国本地数据计算。结局指标包括质量调整生命年(QALY)、生命年(LY)、总成本、增量成本效用比和增量成本效果比(ICUR、ICER),并进行概率敏感性分析(PSA)评估结果稳健性。**结果:**liso-cel治疗获得5.7 QALY(6.4 LY),成本为265,907;SOC获得4.4 QALY(5.1 LY),成本为233,903。与SOC相比,ICUR估计为每QALY 24,580,ICER为每增加一个生命年23,464。PSA显示,在92%的模拟中,liso-cel疗效更高且成本也更高。**结论:**对于法国复发/难治性LBCL患者,liso-cel相较SOC具有成本效果。仍需生成长期真实世界数据以验证这些发现。
CAR-T cell therapies such as lisocabtagene maraleucel (liso-cel) have transformed the treatment of patients with second line primary refractory or early relapsed 12 months (R/R) large B-cell lymphoma (LBCL). The objective of this study was to assess the cost-effectiveness of liso-cel compared to standard of care (SOC) to treat R/R LBCL in France.
A 3-health-state model was developed to compare liso-cel to SOC over 20 years in France. Efficacy and safety were extrapolated from the TRANSFORM trial (NCT03575351), or DESCAR-T (registry of patients treated with a CAR-T in France). Costs were calculated using French-specific sources. Outcomes were quality-adjusted life years (QALY), life-years (LY), total costs, incremental cost-utility and cost-effectiveness ratios (ICUR, ICER). Probabilistic sensitivity analysis (PSA) was conducted to assess the robustness of the results.
Liso-cel generated 5.7 QALY (6.4 LY) for a 265 907. SOC generated 4.4 QALY (5.1 LY) for 233 903. ICUR and ICER were estimated at 24 580/QALY and 23 464/LY gained versus SOC. PSA showed that liso-cel was more effective and more costly in 92% of the simulations.
Liso-cel is cost-effective versus SOC to treat R/R LBCL patients in France. Generation of long-term real-world data is needed in order to validate these findings.
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