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美国弥漫大 B 细胞淋巴瘤治疗路径的成本效果分析

英文原题:A Cost-Effectiveness Analysis of Diffuse Large B-Cell Lymphoma Treatment Pathways in the United States.

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A Cost-Effectiveness Analysis of Diffuse Large B-Cell Lymphoma Treatment Pathways in the United States.

PubMed 2025/06/25(内容时间) MDM Policy Pract Q3 · IF 1.9(JCR 2025)

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中文摘要

背景。嵌合抗原受体(CAR)T细胞疗法已被批准作为弥漫性大B细胞淋巴瘤(DLBCL)的二线(2L)或更后线治疗。近期,双特异性T细胞抗体(BsAbs)已被批准作为三线(3L)治疗。不同治疗序列的成本效益尚不清楚。

本研究旨在从美国医疗保健视角,在每质量调整生命年(QALY)150,000美元的成本效益阈值下,评估axicabtagene ciloleucel(axi-cel)与其他2L DLBCL治疗方案相比的成本效益。设计。本经济学评价使用了离散事件模拟决策。模型输入来自8项临床试验和已发表文献。模拟患者接受2L axi-cel后接受3L治疗,并将其与2L意向性自体干细胞移植(ASCT)、polatuzumab vedotin联合bendamustine和rituximab(Pola-BR)、tafasitamab联合lenalidomide(tafa-len)或rituximab联合gemcitabine和oxaliplatin(R-GemOx)的治疗序列进行比较,所有这些方案随后均接受3L治疗(挽救性化疗、BsAbs或axi-cel)。

此外,还在3L中将axi-cel与glofitamab和epcoritamab直接比较。成本和QALY按3.0%贴现,用于得出增量成本效益比(ICER)和净货币收益(NMB)。结果。在2L基础病例中,axi-cel与意向性ASCT相比具有成本效益(ICER $145,004/QALY),而意向性ASCT与R-GemOx相比具有成本效益(ICER $9,495/QALY)。在$150,000和$200,000/QALY阈值下,axi-cel使NMB最大化,而在$100,000/QALY阈值下,意向性ASCT使NMB最大化。在针对3L的与epcoritamab和glofitamab的比较中,axi-cel分别具有优势和成本效益(ICER $122,224/QALY)。

Axi-cel在$150,000和$200,000/QALY阈值下使NMB最大化,而glofitamab在$100,000/QALY阈值下使NMB最大化。结论。研究结果表明,尽管其他治疗在较低阈值下具有成本效益,axi-cel在美国2L/3L治疗环境中是一种具有成本效益的治疗选择。亮点:本研究探讨了axicabtagene ciloleucel(axi-cel)在当前复发/难治性(R/R)弥漫性大B细胞淋巴瘤(DLBCL)治疗模式中,在二线(2L)和三线(3L)治疗序列中是否具有成本效益。使用一种新型治疗序列模型,发现axi-cel在2L治疗序列中以及与3L双特异性T细胞抗体的直接比较中均具有成本效益。这些发现表明,无论治疗线位置如何,axi-cel都是R/R DLBCL的一种具有成本效益的治疗方法。

展开英文摘要原文

UNLABELLED: Background. Chimeric antigen receptor (CAR) T-cell therapies are approved as second-line (2L) or later therapy for diffuse large B-cell lymphoma (DLBCL). Recently, bispecific T-cell antibodies (BsAbs) have been approved as third-line (3L) treatments. The cost-effectiveness of different treatment sequences is unknown.

This study aims to evaluate the cost-effectiveness of axicabtagene ciloleucel (axi-cel) compared with other treatment options for 2L DLBCL, from a US health care perspective at a cost-effectiveness threshold of $150,000 per quality-adjusted life-year (QALY). Design. This economic evaluation used a discrete event simulation decision. Model inputs were derived from 8 clinical trials and the published literature.

Simulated patients received 2L axi-cel followed by 3L treatments, which were compared with treatment sequences of 2L intended autologous stem cell transplant (ASCT), polatuzumab vedotin with bendamustine and rituximab (Pola-BR), tafasitamab with lenalidomide (tafa-len), or rituximab with gemcitabine and oxaliplatin (R-GemOx), all of which were followed by 3L treatments (salvage chemotherapy, BsAbs, or axi-cel).

In addition, axi-cel was compared directly with glofitamab and epcoritamab in 3L. Costs and QALYs, discounted at 3. 0%, were used to derive incremental cost-effectiveness ratios (ICERs) and net monetary benefits (NMBs). Results. In the 2L base case, axi-cel was cost-effective compared with intended ASCT (ICER $145,004/QALY), which was cost-effective compared with R-GemOx (ICER $9,495/QALY). Axi-cel maximized NMB at $150,000 and $200,000/QALY thresholds, whereas intended ASCT maximized NMB at $100,000/QALY. In 3L-focused comparisons with epcoritamab and glofitamab, axi-cel was dominant and cost-effective (ICER $122,224/QALY), respectively.

Axi-cel maximized NMB at $150,000 and $200,000/QALY thresholds, whereas glofitamab maximized NMB at $100,000/QALY. Conclusions. The findings of the study suggest that although other treatments were cost-effective at lower thresholds, axi-cel is a cost-effective treatment option in 2L/3L settings in the United States.

HIGHLIGHTS: This study investigated whether axicabtagene ciloleucel (axi-cel) is cost-effective in second-line (2L) and third-line (3L) treatment sequences in the current relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) treatment paradigm. Using a novel treatment sequencing model, axi-cel was found to be cost-effective in both 2L treatment sequences and in direct comparisons with 3L bispecific T-cell antibodies.

These findings suggest that axi-cel is a cost-effective treatment for R/R DLBCL regardless of treatment line positioning.

论文信息

作者
Patel AR、Kievit B、Hasegawa K、Ray M、Sharma R、Hofmann S、Blissett R、Locke FL
第一作者单位
Kite, a Gilead Company, Santa Monica, CA, USA.United States
通讯作者单位
H. Lee Moffitt Cancer Center & Research Institute, Tampa, USA.United States
期刊
MDM policy & practice2025 Jan-Jun
原文标识
PubMed 40575250 · DOI 10.1177/23814683251345780