Vanderbilt-Ingram Cancer Center, Nashville, TN, USA.
OPEN Health Company, Bethesda, MD, USA.
J Med Econ. 2022 Jan-Dec;25(1):541-551. doi: 10.1080/13696998.2022.2065787.
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.
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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