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在美国,阿基仑赛二线治疗大 B 细胞淋巴瘤的成本效益:ZUMA-7 试验的经济学评价。

The Cost-Effectiveness of Axicabtagene Ciloleucel as Second-Line Therapy in Patients with Large B-Cell Lymphoma in the United States: An Economic Evaluation of the ZUMA-7 Trial.

机构信息

Memorial Sloan Kettering Cancer Center, New York, New York.

Princess Margaret Cancer Centre, University of Toronto, Toronto, Canada.

出版信息

Transplant Cell Ther. 2022 Nov;28(11):750.e1-750.e6. doi: 10.1016/j.jtct.2022.08.010. Epub 2022 Aug 12.

Abstract

Axicabtagene ciloleucel (axi-cel) was found to have superior clinical outcomes compared to standard of care (SOC; salvage chemoimmunotherapy, followed by high-dose therapy with autologous stem cell rescue for responders) for second-line large B-cell lymphoma (2L LBCL) in the pivotal ZUMA-7 trial. The aim of this analysis was to evaluate the cost effectiveness of using axi-cel compared to the current standard 2L LBCL therapy. A 3-state partitioned-survival model estimated the cost effectiveness and budget impact from a payer perspective in the United States. Clinical outcomes were extrapolated based on the pivotal trial. The model calculated expected quality-adjusted life years (QALYs), total costs (in United States dollars [USD], and the incremental cost-effectiveness ratio (ICER), along with the budget impact. Sensitivity and scenario analyses were performed. The proportion alive at 10 years was estimated as 48% for axi-cel and 38% for SOC; median overall survival was estimated at 59 and 24 months for axi-cel and SOC, respectively. Over a lifetime horizon, the model estimated a total of 5.56 and 7.08 QALYs for SOC and axi-cel, respectively, of which 41% and 74% were in the event-free state, respectively. Incremental QALYs and costs were 1.51 and $100,366 USD, resulting in an ICER of $66,381 USD per QALY for axi-cel versus SOC. Despite crossover to subsequent CAR T in the SOC arm, second-line CAR T use was found to improve the quality and length of life compared to SOC. Cost offsets due to subsequent CAR T use led to a limited incremental cost difference. Treatment with axi-cel is a cost-effective option that addresses an important unmet clinical need for patients with LBCL who relapse or are refractory to front-line therapy.

摘要

阿基仑赛(axi-cel)在关键的 ZUMA-7 试验中与标准治疗(SOC;二线大 B 细胞淋巴瘤(2L LBCL)的挽救性化疗免疫治疗,随后对缓解者进行大剂量治疗和自体干细胞挽救)相比,具有优越的临床结果。本分析旨在评估与当前标准 2L LBCL 治疗相比,使用 axi-cel 的成本效益。一个 3 状态分区生存模型从支付者的角度评估了美国使用 axi-cel 的成本效益和预算影响。根据关键试验推断临床结果。该模型计算了预期的质量调整生命年(QALY)、总成本(以美元计)和增量成本效益比(ICER),以及预算影响。进行了敏感性和情景分析。估计 axi-cel 的 10 年生存率为 48%,SOC 为 38%;中位总生存期估计分别为 axi-cel 和 SOC 的 59 和 24 个月。在终身时间范围内,模型估计 SOC 和 axi-cel 的总 QALY 分别为 5.56 和 7.08,其中分别有 41%和 74%处于无事件状态。增量 QALY 和成本分别为 1.51 和 100366 美元,导致 axi-cel 相对于 SOC 的每 QALY 增量成本效益比为 66381 美元。尽管 SOC 臂中存在交叉到随后的 CAR T,但二线 CAR T 的使用被发现可提高生活质量和延长寿命,优于 SOC。由于随后的 CAR T 使用而导致的成本抵消导致增量成本差异有限。对于接受一线治疗后复发或耐药的 LBCL 患者,axi-cel 的治疗是一种具有成本效益的选择,可满足重要的未满足的临床需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/593f/10314822/bb72d87201d7/nihms-1908318-f0001.jpg

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