肿瘤细胞治疗研究
英文原题：Chimeric antigen receptor-natural killer cell therapy in first-relapse epithelial ovarian cancer: an early health-economic evaluation in the Netherlands.
CAR-NK 细胞疗法对铂敏感 EOC 可能具有成本效益。
目的：通过早期卫生经济学建模，评估在荷兰为首次复发的铂类敏感、高级别上皮性卵巢癌（EOC）患者，在标准治疗（SOC）基础上增加 CAR-NK（嵌合抗原受体自然杀伤）细胞疗法的潜在成本效果。 方法：从荷兰医疗系统角度建立分区生存模型，采用终身时间范围和 3 周周期，设定无进展、复发疾病和死亡健康状态。按突变状态（BRCA、同源重组缺陷或无突变）区分治疗方案，将各 SOC 方案与相同方案加重复 CAR-NK 输注进行比较。分析两种 CAR-NK 治疗费用情景：每周期 15,000 和 20,000（原摘要货币符号缺失）。成本和效果按每年 3% 和 1.5% 折现。进行情景和多变量敏感性分析，成本效果阈值为每质量调整生命年（QALY）80,000（原文货币符号缺失）。 结果：SOC 策略每位患者产生 2.47 个 QALY 和 3.2 个生命年，成本为 34,340（原文货币符号缺失）。CAR-NK 策略需额外获得 0.76–0.95 个 QALY（1.00–1.25 个生命年）才能达到成本效果，相当于无进展生存期中位数增加 3.08 个月、总生存期增加 10.82–13.60 个月。成本效果对 CAR-NK 细胞治疗费用、生存获益和效用值最敏感。 结论：CAR-NK 细胞疗法有望成为铂类敏感 EOC 的成本有效治疗，但需带来显著总生存获益。早期经济学建模可在不确定性下为开发提供策略指导、识别价值驱动因素并帮助设计临床试验。
OBJECTIVE: To use early health economic modelling to evaluate the potential cost-effectiveness of adding a CAR-NK (chimeric antigen receptor-natural killer) cell therapy to standard of care (SOC) in patients with platinum-sensitive, high-grade epithelial ovarian cancer (EOC) at first recurrence in the Netherlands. METHODS: A partitioned survival model was developed from the Dutch healthcare perspective with a lifetime horizon and 3-week cycles, including progression-free, recurrent-disease and dead health states. Treatment regimens, varying by mutation status (BRCA, homologous recombination deficiency or no mutation), were compared with the same regimen plus repeated CAR-NK cell infusion (CAR-NK strategy). Two cost scenarios ( 15 000 and 20 000 per CAR-NK cell therapy cycle) were analyzed. Costs and effects were discounted at 3% and 1.5% annually. Scenario and multivariate sensitivity analyses were conducted. A cost-effectiveness threshold of 80 000 per QALY was applied. RESULTS: The SoC strategy yielded 2.47 QALYs and 3.2 life-years at 34 340 per patient. CAR-NK strategy would require 0.76-0.95 additional QALYs (1.00-1.25 life-years) to be cost-effective. This corresponds to median gains of 3.08 months in progression-free survival and 10.82-13.60 months in overall survival. Cost-effectiveness was most sensitive to CAR-NK cell therapy costs, survival gains and utility values. CONCLUSIONS: A CAR-NK cell therapy could become cost-effective for platinum-sensitive EOC. To achieve this, it would need to deliver substantial overall survival gains. Early economic modeling provides strategic guidance for development, identifies key drivers of value and informs clinical trial design under uncertainty.
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