肿瘤细胞治疗研究
英文原题：Economic Evaluation of Axicabtagene Ciloleucel as a Second-Line Therapy for Relapsed/Refractory Diffuse Large B-Cell Lymphoma in South Korea: A Cost-Utility Analysis Based on the ZUMA-7 Trial.
Economic Evaluation of Axicabtagene Ciloleucel as a Second-Line Therapy for Relapsed/Refractory Diffuse Large B-Cell Lymphoma in South Korea: A Cost-Utility Analysis Based on the ZUMA-7 Trial.
Axi-cel作为复发或难治性DLBCL的二线治疗，在生存期和质量调整生命年方面提供了有意义的获益，与韩国SoC相比，似乎是一种具有成本效益的替代方案。
弥漫性大B细胞淋巴瘤（DLBCL）是韩国最常见的非霍奇金淋巴瘤亚型，30-40%的患者在一线治疗后复发或难治。在ZUMA-7试验中，axicabtagene ciloleucel（axi-cel）显示出优于标准治疗（SoC）挽救性化疗联合自体干细胞移植（ASCT）的疗效。虽然axi-cel已在韩国获批用于二线治疗，但尚未在当地医疗环境中评估其成本效果。本研究旨在从韩国医疗系统的角度，评估axi-cel与SoC相比，作为适合ASCT的复发或难治性DLBCL成人患者二线治疗的成本效果。
基于ZUMA-7数据开发了一个采用混合治愈方法的分区生存模型，用于比较axi-cel与SoC作为适合移植的复发/难治性DLBCL二线治疗的效果。该分析从医疗体系角度出发，采用终身时间 horizon，成本和结果按每年4.5%贴现。效用值根据ZUMA-7中的EQ-5D-5L数据，使用韩国估值集进行估计。成本包括治疗、后续治疗、资源使用、不良事件管理和临终关怀。进行了确定性和概率敏感性分析。成本效果使用韩国人均国内生产总值的1.5-2倍作为支付意愿阈值进行评估。
在终生时间范围内，axi-cel 产生了 8.68 生命年（LYs）和 7.61 质量调整生命年（QALYs），而 SoC 为 7.10 LYs 和 5.91 QALYs。axi-cel 的总成本更高，导致增量成本为 9390 万韩元（68,676 美元（USD））。增量成本效果比（ICER）为每获得一个 QALY 5550 万韩元（40,583 美元）。敏感性分析显示结果稳健，后续治疗模式是最具影响力的参数。
BACKGROUND AND OBJECTIVES: Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin lymphoma in South Korea, with 30-40% of patients relapsing or becoming refractory after first-line therapy. Axicabtagene ciloleucel (axi-cel) demonstrated superior efficacy over standard-of-care (SoC) salvage chemotherapy plus autologous stem cell transplantation (ASCT) in the ZUMA-7 trial. While approved for second-line use in South Korea, its cost effectiveness has not yet been evaluated in the local healthcare setting. This study aimed to evaluate the cost effectiveness of axi-cel compared with SoC as a second-line treatment for adult patients with relapsed or refractory DLBCL eligible for ASCT from the perspective of the South Korean healthcare system. METHODS: A partitioned survival model with a mixture cure approach was developed based on ZUMA-7 data to compare axi-cel with SoC as second-line treatment for transplant-eligible relapsed/refractory DLBCL. The analysis was conducted from the healthcare system perspective over a lifetime horizon, with costs and outcomes discounted at 4.5% annually. Utilities were estimated from EQ-5D-5L data in ZUMA-7 using the South Korean valuation set. Costs included treatment, subsequent therapies, resource use, adverse event management, and end-of-life care. Deterministic and probabilistic sensitivity analyses were conducted. Cost effectiveness was evaluated using a willingness-to-pay threshold of 1.5-2 times the gross domestic product per capita of South Korea. RESULTS: Over a lifetime horizon, axi-cel yielded 8.68 life years (LYs) and 7.61 quality-adjusted life years (QALYs) versus 7.10 LYs and 5.91 QALYs for SoC. Total costs were higher for axi-cel, resulting in an incremental cost of KRW 93.9 million (US dollars (USD) 68,676). The incremental cost-effectiveness ratio (ICER) was KRW 55.5 million (USD 40,583) per QALY gained. Sensitivity analyses showed robust results, with subsequent therapy pattern as the most influential parameter. CONCLUSIONS: Axi-cel as second-line treatment for relapsed or refractory DLBCL provided meaningful gains in survival and quality-adjusted life and appeared to be a cost-effective alternative compared to SoC in South Korea.
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