肿瘤细胞治疗研究
英文原题：Cost-Effectiveness Analysis of Daratumumab Monotherapy and Subsequent Therapies in Heavily Treated Relapsed/Refractory Multiple Myeloma: A Feasible Methodology using a Korean Nationwide Population Cohort.
Cost-Effectiveness Analysis of Daratumumab Monotherapy and Subsequent Therapies in Heavily Treated Relapsed/Refractory Multiple Myeloma: A Feasible Methodology using a Korean Nationwide Population Cohort.
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本研究提供了一个新的成本效果框架，将治疗效果与现实世界成本联系起来，支持对未来 MM 疗法的社会成本进行预测。
多发性骨髓瘤（MM）的高成本新型疗法需要评估其疗效和成本效益。
本研究开发了一种利用全国11450例新诊断MM患者数据评估成本效果的方法学。应用了一种新算法来识别治疗线数（LoT）。
新诊断MM患者数量显著增加，从2010年的873例增至2019年的1,464例（p < 0.001）。LoT越晚，至下次治疗时间（TTNT）和总生存期（OS）越短（p < 0.001），而全因医疗费用随每个LoT增加（p < 0.001）。硼替佐米-美法仑-泼尼松龙是不适合移植患者最常用的一线方案（29.2%），而硼替佐米-沙利度胺-地塞米松是适合移植患者最常用的方案（11.3%）。在重度治疗的MM患者中，与标准治疗相比，达雷妥尤单抗单药治疗显示出更优的第二TTNT（7.8 vs. 5.2个月）和OS（8.5 vs. 5.3个月），且在成本调整后仍保持统计学显著性。对于达雷妥尤单抗后的后续治疗，开发了一种使用增量成本效果比（ICER）估算所需医疗费用的方法：预期成本（$）=ICER×（预期寿命-0.567）+35,601。
High-cost novel therapies for multiple myeloma (MM) require evaluation of efficacy and cost-effectiveness.
This study developed a methodology to assess cost-effectiveness using nationwide data from 11,450 newly diagnosed MM patients. A novel algorithm was applied to identify lines of therapy (LoT).
The number of newly diagnosed MM patients increased significantly, from 873 in 2010 to 1,464 in 2019 (p < 0.001). Advancing LoT was associated with shorter time to next treatment (TTNT) and overall survival (OS) (p < 0.001), while all-cause medical costs increased with each LoT (p < 0.001). Bortezomib-melphalan-prednisolone was the most common frontline regimen for transplant-ineligible patients (29.2%), while bortezomib-thalidomide-dexamethasone was most used for transplant-eligible patients (11.3%). Daratumumab monotherapy demonstrated superior second TTNT (7.8 vs. 5.2 months) and OS (8.5 vs. 5.3 months) compared to standard care in heavily treated MM patients, with statistical significance maintained after cost adjustment. For subsequent therapies following daratumumab, a methodology was developed to estimate required medical costs using the incremental cost-effectiveness ratio (ICER): Expected cost ($)=ICER×(Expected life expectancy-0.567)+35,601.
This study provides a novel cost-effectiveness framework linking treatment efficacy and real-world costs, supporting predictions of societal costs for future MM therapies.
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