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Eur J Cancer. 2019 Sep;119:18-29. doi: 10.1016/j.ejca.2019.06.011. Epub 2019 Aug 8.
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Pembrolizumab versus ipilimumab in advanced melanoma (KEYNOTE-006): post-hoc 5-year results from an open-label, multicentre, randomised, controlled, phase 3 study.帕博利珠单抗对比伊匹单抗用于晚期黑色素瘤(KEYNOTE-006):一项开放标签、多中心、随机、对照、III 期研究的 5 年随访后结果。
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J Med Econ. 2019 Dec;22(12):1243-1252. doi: 10.1080/13696998.2019.1635487. Epub 2019 Aug 4.
6
Cost-effectiveness of pembrolizumab for the adjuvant treatment of resected high-risk stage III melanoma in the United States.帕博利珠单抗用于美国高风险 III 期黑色素瘤辅助治疗的成本效果分析。
J Med Econ. 2019 Oct;22(10):981-993. doi: 10.1080/13696998.2019.1609485. Epub 2019 May 17.
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Cutaneous Melanoma, Version 2.2019, NCCN Clinical Practice Guidelines in Oncology.皮肤黑色素瘤临床实践指南(第 2 版).2019,NCCN 肿瘤学临床实践指南。
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基因表达谱分析和辅助治疗对 IIIA 期黑色素瘤的成本效益影响。

Cost-Benefit Implication of Gene Expression Profiling and Adjuvant Therapy in Stage IIIA Melanoma.

机构信息

Department of Surgery/Division of Surgical Oncology, Memorial Sloan-Kettering Cancer Center, New York, NY.

Center for Health Policy and Outcomes, Memorial Sloan-Kettering Cancer Center, New York, NY.

出版信息

J Am Coll Surg. 2020 Nov;231(5):547-554.e1. doi: 10.1016/j.jamcollsurg.2020.08.750. Epub 2020 Sep 1.

DOI:10.1016/j.jamcollsurg.2020.08.750
PMID:32889093
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7854824/
Abstract

BACKGROUND

Indiscriminate use of adjuvant therapy in stage IIIA melanoma is controversial. We sought to model the clinical impact and cost of implementing a gene expression profile (GEP) test to guide adjuvant therapy.

STUDY DESIGN

A Markov decision-analysis model was created to represent resected stage IIIA melanoma with 3 treatment options: observation (OBS), adjuvant pembrolizumab for all patients (ALL), and selective adjuvant therapy (SEL). In the SEL option, only high-risk patients based on GEP stratification were treated with pembrolizumab. Cost of adjuvant therapy was normalized to reflect Medicare reimbursement schedules. The primary outcome was cost per mortality avoided at 10 years.

RESULTS

Model projections for 10-year overall survival were 68% for OBS, 73% for SEL, and 76% for ALL. The estimated incremental cost-per-mortality-avoided (compared to OBS) was $2.1 million for SEL and $2.4 million for ALL. These translate to costs of $583.0K and $697.1K per life-year for the SEL and ALL strategies, respectively.

CONCLUSIONS

Routine adjuvant pembrolizumab for stage IIIA melanoma is costly, and risk-stratification by GEP only marginally improves the value of therapy.

摘要

背景

在 IIIA 期黑色素瘤中不加选择地使用辅助治疗存在争议。我们试图建立一个模型,以评估实施基因表达谱(GEP)检测来指导辅助治疗的临床影响和成本。

研究设计

创建了一个 Markov 决策分析模型来代表接受手术治疗的 IIIA 期黑色素瘤患者,有 3 种治疗选择:观察(OBS)、所有患者辅助使用 pembrolizumab(ALL)和选择性辅助治疗(SEL)。在 SEL 选择中,只有基于 GEP 分层的高危患者才接受 pembrolizumab 治疗。辅助治疗的成本进行了归一化处理,以反映医疗保险报销计划。主要结果是 10 年内每避免 1 例死亡的成本。

结果

模型预测 10 年总生存率为 OBS 组 68%、SEL 组 73%和 ALL 组 76%。与 OBS 相比,SEL 组避免 1 例死亡的增量成本为 210 万美元,ALL 组为 240 万美元。这意味着 SEL 和 ALL 策略的每例生命年成本分别为 58.3 万美元和 69.71 万美元。

结论

常规使用 pembrolizumab 辅助治疗 IIIA 期黑色素瘤成本高昂,而通过 GEP 进行风险分层仅略微提高了治疗的价值。