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在美国,对于经过二线或多线系统性治疗后复发或难治性大 B 细胞淋巴瘤的成年患者,与 lisocabtagene maraleucel 相比,axicabtagene ciloleucel 的成本效益。

Cost-effectiveness of axicabtagene ciloleucel versus lisocabtagene maraleucel for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy in the US.

机构信息

Vanderbilt-Ingram Cancer Center, Nashville, TN, USA.

OPEN Health Company, Bethesda, MD, USA.

出版信息

J Med Econ. 2022 Jan-Dec;25(1):541-551. doi: 10.1080/13696998.2022.2065787.

Abstract

AIMS

This study evaluated from a US payer perspective the cost-effectiveness of two chimeric antigen receptor T (CAR T) cell therapies, axicabtagene ciloleucel (axi-cel) versus lisocabtagene maraleucel (liso-cel), for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma (LBCL) following two or more systemic therapy lines.

METHODS

We developed a 3-state (i.e., pre-progression, post-progression, death) partitioned survival model to estimate patients' lifetime outcomes. Mixture cure models were used for survival extrapolation to account for long-term remission. Survival inputs were based on a matching-adjusted indirect comparison (MAIC) that reweighted the ZUMA-1 population (receiving axi-cel) to match patient characteristics in TRANSCEND-NHL-001 (assessing liso-cel). Costs included apheresis, drug acquisition, and administration for conditioning chemotherapy and CAR T therapies, monitoring, transplant, hospitalization, adverse events, routine care, and terminal care, per published literature and databases. Utilities were derived from ZUMA-1 and literature. Deterministic and probabilistic sensitivity analyses were conducted.

RESULTS

In the base case, axi-cel was associated with more QALYs (7.76 vs. 5.94) and greater costs overall ($611,440 vs. $597,174) than liso-cel, at $7,843/QALY gained. The incremental costs (+$14,266) were largely driven by higher routine care costs (+$18,596) due to longer survival and hospitalization (+$10,993) but partially offset by reduced costs of CAR T acquisition (‒$11,300) and terminal care (‒$4,025). Sensitivity analyses consistently suggested robustness of base-case results.

LIMITATIONS

This study relied on an MAIC in which trial design differences and unobserved confounders could not be accounted for. Future real-world studies for recently approved CAR T are warranted to validate our results. Due to a lack of data, we assumed equivalent use of transplants and treatment for B-cell aplasia between the two therapies based on clinicians' opinions.

CONCLUSIONS

In the US, axi-cel is a potentially cost-effective treatment option compared with liso-cel for adult patients with r/r LBCL after two or more systemic therapy lines.

摘要

目的

本研究从美国支付者的角度评估了两种嵌合抗原受体 T(CAR T)细胞疗法——axicabtagene ciloleucel(axi-cel)与 lisocabtagene maraleucel(liso-cel)——在治疗二线或以上全身治疗后复发或难治性(r/r)大 B 细胞淋巴瘤(LBCL)的成年患者中的成本效益。

方法

我们开发了一个 3 状态(即前进展、后进展、死亡)分区生存模型,以估计患者的终生结局。混合治愈模型用于生存外推,以考虑长期缓解。生存输入基于匹配调整的间接比较(MAIC),该比较重新加权 ZUMA-1 人群(接受 axi-cel)以匹配 TRANSCEND-NHL-001 中的患者特征(评估 liso-cel)。成本包括根据已发表的文献和数据库,为调理化疗和 CAR T 治疗、监测、移植、住院、不良事件、常规护理和终末护理进行的单采术、药物获取和管理。效用来自 ZUMA-1 和文献。进行了确定性和概率敏感性分析。

结果

在基线情况下,axi-cel 与更多的 QALYs(7.76 比 5.94)和更高的总成本(611440 美元比 597174 美元)相关,而 liso-cel 的增量成本(+14266 美元)主要归因于更长的生存和住院时间导致的常规护理成本增加(+18596 美元),但部分被 CAR T 获得成本降低(-11300 美元)和终末护理成本降低(-4025 美元)所抵消。敏感性分析一致表明,基本情况结果具有稳健性。

局限性

本研究依赖于 MAIC,其中试验设计差异和未观察到的混杂因素无法得到解释。需要进行最近批准的 CAR T 的真实世界研究,以验证我们的结果。由于缺乏数据,我们根据临床医生的意见,假设两种疗法之间用于 B 细胞再生障碍的移植和治疗的等效使用。

结论

在美国,对于二线或以上全身治疗后复发或难治性大 B 细胞淋巴瘤的成年患者,axi-cel 是一种潜在的具有成本效益的治疗选择,优于 liso-cel。

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