为指导中危髓系恶性肿瘤患者的治疗而进行的 37 基因小组与常规护理的成本效用探索性分析。

IF 3.1 4区 医学 Q1 ECONOMICS
Daniel Lindsay, Andrea Henden, Ricky Nelles, Thomas M Elliott, Louisa G Collins
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引用次数: 0

摘要

目的:骨髓恶性肿瘤的基因组风险分层方法已经超越了传统的核型分析和单基因方法,可以更好地界定疾病行为。下一代测序已被确定为新的标准治疗工具,可在诊断时准确确定预后并指导治疗决策。我们的目的是确定 37 个基因全套检测的经济价值,为中危髓系恶性肿瘤患者的后续治疗提供依据:我们对澳大利亚昆士兰州的骨髓恶性肿瘤患者进行了一项探索性成本效用分析,为干细胞移植治疗提供依据。临床医生调查提供了有基因组学信息和无基因组学信息时的治疗选择数据,而医疗成本、生活质量、复发率和存活率数据则采用了公开数据和个人数据。我们利用马尔可夫链决策分析队列模型和 5000 次模拟,得出了每个质量调整生命年(QALY)的增量成本。进行了情景分析、单向分析和概率敏感性分析,以检验输入变量对主要研究结果稳定性的影响:在 10 年内,模型预测小组检测策略的平均成本为 125,561 澳元,常规护理的平均成本为 117,045 澳元,表明小组检测的增量成本为 8516 澳元。相应的平均 QALY 为:面板检测 4.52,常规护理 4.46,每获得一个 QALY 的成本为 153,854 澳元。在澳大利亚的系统中,小组检测具有成本效益的可能性为结论:中危髓系恶性肿瘤患者在接受治疗后的存活率和复发率略有提高,受此影响,在澳大利亚进行基因组测序不太可能具有成本效益。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Exploratory Cost-Utility Analysis of a 37-Gene Panel Versus Usual Care to Guide Therapy for Patients with Intermediate-Risk Myeloid Malignancies.

Objective: Genomic risk stratification methods for myeloid malignancies have moved beyond conventional karyotyping and single gene approaches to better define disease behaviour. Next-generation sequencing has been established as the new standard-of-care tool to accurately define prognosis at diagnosis and guide therapy decisions. We aimed to determine the economic value of a 37-gene panel test for informing subsequent care for patients with intermediate-risk myeloid malignancies.

Method: We performed an exploratory cost-utility analysis of a 37-gene panel test to inform stem cell transplantation therapy in patients with myeloid malignancies in Queensland, Australia. Clinician surveys provided data on management choice with and without genomics information while both published and individual-level data were used for healthcare costs, quality of life, relapse rates and survival data. We used a decision-analytic cohort model with Markov chains and 5000 simulations to derive the incremental cost per quality-adjusted life year (QALY) gained. Scenario, one-way and probabilistic sensitivity analyses were undertaken to test input variation on the stability of the main findings.

Results: Over 10 years, the model predicted mean costs of AU$125,561 for the panel testing strategy and AU$117,045 for usual care, indicating an incremental cost of AU$8516 for panel testing. The corresponding mean QALYs were 4.52 for panel testing and 4.46 for usual care, producing a cost of AU$153,854 per QALY gained. In the Australian system, the likelihood that panel testing would be cost effective was <1 % and would have a more favourable cost-effective profile at a willingness-to-pay of AU$140,000 per QALY gained.

Conclusions: Driven by small gains in survival and relapse rates following therapies, genomic panel sequencing for myeloid malignancies in people with intermediate-risk disease is unlikely to be cost effective in Australia.

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来源期刊
Applied Health Economics and Health Policy
Applied Health Economics and Health Policy Economics, Econometrics and Finance-Economics and Econometrics
CiteScore
6.10
自引率
2.80%
发文量
64
期刊介绍: Applied Health Economics and Health Policy provides timely publication of cutting-edge research and expert opinion from this increasingly important field, making it a vital resource for payers, providers and researchers alike. The journal includes high quality economic research and reviews of all aspects of healthcare from various perspectives and countries, designed to communicate the latest applied information in health economics and health policy. While emphasis is placed on information with practical applications, a strong basis of underlying scientific rigor is maintained.
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