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脂质体伊立替康治疗胰腺腺癌患者的有效性和成本分析:一项多中心真实世界研究

Effectiveness and cost analysis of liposomal irinotecan in patients with pancreatic adenocarcinoma: a multicenter real-world study.

作者信息

Chun Jung Won, Kim Young Ae, Lee Jang Won, Lee Danbi, Jang Dong Kee, Lee Yoon Suk, Lee Jong-Chan, Lee Woo Jin, Hwang Jin-Hyeok, Woo Sang Myung

机构信息

Research Institute and Hospital, National Cancer Center, Goyang, Republic of Korea.

National Cancer Control Institute, National Cancer Center, Goyang, Republic of Korea.

出版信息

Ther Adv Med Oncol. 2025 Sep 13;17:17588359251370492. doi: 10.1177/17588359251370492. eCollection 2025.

Abstract

BACKGROUND

Systemic chemotherapy is crucial for pancreatic adenocarcinoma (PAC). It serves as a palliative chemotherapy for unresectable PAC and as a (neo-)adjuvant chemotherapy for (borderline) resectable PAC. Survival benefits were found with the use of liposomal irinotecan combined with 5-fluorouracil and leucovorin (nal-IRI/5-FU/LV) in the global phase III NAPOLI-1 trial. However, limited evidence supports the cost-effectiveness of liposomal irinotecan throughout the PAC treatment course.

OBJECTIVES

We investigated the real-world efficacy and cost implications of nal-IRI/5-FU/LV in PAC management.

DESIGN

A multicenter real-world study.

METHODS

We analyzed real-world data from electronic health records to evaluate outcomes of nal-IRI/5-FU/LV exposure in this retrospective study. Effectiveness was assessed using overall survival (OS). Medical costs were those incurred from imaging or laboratory tests, chemotherapy, surgery, radiotherapy, and supportive care during inpatient, outpatient, and emergency department visits. Propensity score matching was used to adjust for potential differences in patient characteristics between the nal-IRI and non-liposomal irinotecan groups. The incremental cost-effectiveness ratio (ICER) was calculated by dividing cost differences by survival gains.

RESULTS

Overall, 1734 patients diagnosed with PAC were screened, and 705 who received at least one cycle of chemotherapy were included in the analysis. The median OS was significantly longer in the nal-IRI group than in the non-liposomal irinotecan group (28.0 vs 18.3 months,  = 0.011). Patients in the nal-IRI group had more hospital visits and admissions during follow-up and incurred higher costs ($61,430 vs $39,129 per patient), which resulted in an ICER of $2787 per month of survival gained ($33,285 per life-year gained).

CONCLUSION

Despite its higher cost, nal-IRI/5-FU/LV significantly improved the survival of patients with PAC compared to the conventional chemotherapies. Our findings suggest that it may be a cost-effective treatment option for gemcitabine failure despite the high cost of liposomal irinotecan, considering the poor prognosis of PAC.

摘要

背景

全身化疗对胰腺腺癌(PAC)至关重要。它可作为不可切除PAC的姑息化疗,以及(边缘性)可切除PAC的(新)辅助化疗。在全球III期NAPOLI-1试验中,发现脂质体伊立替康联合5-氟尿嘧啶和亚叶酸钙(nal-IRI/5-FU/LV)可带来生存获益。然而,支持脂质体伊立替康在整个PAC治疗过程中成本效益的证据有限。

目的

我们调查了nal-IRI/5-FU/LV在PAC治疗中的实际疗效和成本影响。

设计

一项多中心真实世界研究。

方法

在这项回顾性研究中,我们分析了来自电子健康记录的真实世界数据,以评估nal-IRI/5-FU/LV暴露的结果。使用总生存期(OS)评估有效性。医疗成本包括住院、门诊和急诊科就诊期间的影像或实验室检查、化疗、手术、放疗及支持治疗所产生的费用。采用倾向评分匹配法来调整nal-IRI组和非脂质体伊立替康组患者特征的潜在差异。通过将成本差异除以生存获益来计算增量成本效益比(ICER)。

结果

总体而言,筛查了1734例诊断为PAC的患者,其中705例接受了至少一个周期化疗的患者纳入分析。nal-IRI组的中位OS显著长于非脂质体伊立替康组(28.0个月对18.3个月,=0.011)。nal-IRI组患者在随访期间有更多的医院就诊和住院次数,且产生了更高的费用(每位患者61,430美元对39,129美元),这导致每获得一个月生存期的ICER为2787美元(每获得一个生命年为33,285美元)。

结论

尽管成本较高,但与传统化疗相比,nal-IRI/5-FU/LV显著提高了PAC患者的生存率。我们的研究结果表明,考虑到PAC的预后较差,尽管脂质体伊立替康成本高昂,但对于吉西他滨治疗失败的患者,它可能是一种具有成本效益的治疗选择。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e63b/12433562/bc0574bc1fa7/10.1177_17588359251370492-fig1.jpg

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