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 CAR T-Cell Therapies vs Antibody Drug Conjugates for Patients with Advanced Multiple Myeloma.
Cost-Effectiveness Analysis of CAR T-Cell Therapies vs Antibody Drug Conjugates for Patients with Advanced Multiple Myeloma.
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这些发现依赖于长期生存的假设,但表明 CAR-T 疗法的使用正接近标准的 ICER 阈值。
在晚期多发性骨髓瘤(MM)患者中,B细胞成熟抗原(BCMA)特异性靶点如Belantamab Mafodotin(belamaf)和CAR-T 细胞疗法已被证明可改善临床结局,但成本高昂。为了比较一个假设性三重难治性MM患者队列中,接受三种BCMA靶向治疗之一所获得的每质量调整生命年(QALYs)的预期成本:(1)idecabtagene vicleucel(ide-cel),(2)ciltacabtagene autoleucel(cilta-cel),以及(3)belamaf长达20个月。
在这项成本效果分析中,我们利用MM治疗证据中的估计值和参数,构建了一个蒙特卡洛马尔可夫链微观模拟模型,针对10000名年龄在40至80岁之间的假设患者。我们分配了预期剩余生命年数,并对5年后的生存情况做出了不同的假设。
我们预测CAR-T 疗法的总治疗费用是belamaf的六倍,但治疗获得的QALYs却高出6至8倍。ide-cel被cilta-cel弱支配,我们比较cilta-cel与belamaf的基准案例增量成本效果比(ICER)为每获得一个QALY需109,497美元,在概率敏感性分析中平均为123,618美元。
Among advanced multiple myeloma (MM) patients, B-cell maturation antigen (BCMA) specific targets like Belantamab Mafodotin (belamaf) and CAR T-cell therapies have been shown to improve clinical outcomes, but at significant costs. To compare the expected costs per quality-adjusted life years (QALYs) gained among a hypothetical cohort of triple refractory MM patients treated with one of three BCMA-directed therapies: (1) idecabtagene vicleucel (ide-cel), (2) ciltacabtagene autoleucel (cilta-cel), and (3) belamaf for up to 20 months.
In this cost-effectiveness analysis, we built a Monte Carlo Markov Chain microsimulation model using estimates and parameters from the evidence on MM treatment for 10 000 hypothetical patients between the ages for 40 and 80. We assigned expected years of life remaining and made varying assumptions about survival beyond 5 years.
We predicted total cost of treatment for CAR-T therapy to be six times greater than for belamaf, but the QALYs gained from treatment are 6 to 8 times greater. Ide-cel was weakly dominated by cilta-cel and our base-case incremental cost effectiveness ratio (ICER) comparing cilta-cel with belamaf was $109,497 per QALY gained, averaging $123,618 in probabilistic sensitivity analyses.
These findings hinge on the assumption of longer-term survival but suggest that the use of CAR-T therapy is approaching standard ICER thresholds.
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