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评估正电子发射断层扫描/计算机断层扫描(PET/CT)用于肿瘤治疗反应监测的成本效益的建模方法的系统文献综述

A Systematic Literature Review of Modelling Approaches to Evaluate the Cost Effectiveness of PET/CT for Therapy Response Monitoring in Oncology.

作者信息

van Mossel Sietse, de Feria Cardet Rafael Emilio, de Geus-Oei Lioe-Fee, Vriens Dennis, Koffijberg Hendrik, Saing Sopany

机构信息

Department of Radiology, Leiden University Medical Centre, Leiden, The Netherlands.

Biomedical Photonic Imaging Group, University of Twente, PO Box 217, 7500 AE, Enschede, The Netherlands.

出版信息

Pharmacoeconomics. 2025 Feb;43(2):133-151. doi: 10.1007/s40273-024-01447-y. Epub 2024 Nov 3.

Abstract

BACKGROUND AND OBJECTIVE

This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature.

METHODS

This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications.

RESULTS

The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias.

CONCLUSIONS

Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addressed appropriately. Further research should include more advanced and patient-level modelling approaches to accurately represent the complex context of clinical practice and, therefore, to be meaningful to support decision making.

REGISTRATION

This review is registered in PROSPERO, the international prospective register of systematic reviews funded by the National Institute for Health Research, with CRD42023402581.

摘要

背景与目的

本系统文献综述探讨了基于模型的成本效益研究,该研究用于通过正电子发射断层扫描(PET)监测治疗反应,PET通常与低剂量计算机断层扫描(CT)结合用于各种癌症类型。鉴于已知治疗反应事件存在异质性,研究应考虑患者层面的建模而非基于队列的建模,因为其在处理这些事件及事件发生时间方面具有灵活性。本综述旨在确定所使用的建模方法,并对当前文献中所做的假设进行系统评估。

方法

本研究按照系统评价与Meta分析的首选报告项目(PRISMA)2020声明进行实施和报告。信息来源包括电子书目数据库、综述文章的参考文献列表以及与核医学、卫生技术评估和卫生经济学领域专家的联系。纳入标准包括同行评审的科学出版物和已发表的灰色文献。文献检索、筛选和批判性评价由两名评审员独立进行。采用综合卫生经济评价报告标准(CHEERS)评估方法学质量。使用经济评价中的偏倚(ECOBIAS)清单来确定纳入出版物中的偏倚风险。

结果

检索结果包括2959篇出版物。纳入数据提取和综合的出版物数量为10篇,代表8项独特研究。这些研究涉及淋巴瘤、晚期头颈癌、脑肿瘤、非小细胞肺癌和宫颈癌患者。所有研究均涉及对化疗的反应。没有研究评估对免疫疗法的反应。大多数研究将PET/CT定位为附加模式,一项研究将PET/CT定位为传统成像(X射线和增强CT)的替代方法。三项研究报告了决策树结构,四项研究报告了队列层面的状态转换模型,一项研究报告了分区生存模型。没有报告患者层面的模型。采用的模拟期从1年到终身不等。大多数研究报告了概率分析,而两项研究仅报告了确定性分析。两项研究进行了信息价值分析。多项研究未充分讨论模型特定的偏倚方面。最重要且经常观察到的是结构假设偏倚、有限模拟期偏倚和错误模型偏倚的高风险。

结论

在当前文献中,基于模型的PET/CT治疗反应监测成本效益分析是基于患者队列而非个体患者。因此,治疗反应事件中的异质性通常未得到适当处理。进一步的研究应包括更先进的患者层面建模方法,以准确反映临床实践中的复杂情况,从而对支持决策具有意义。

注册情况

本综述已在由英国国家卫生研究院资助的国际系统评价前瞻性注册库PROSPERO中注册,注册号为CRD42023402581。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9ef8/11782410/1e73aa431c2b/40273_2024_1447_Fig1_HTML.jpg

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