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使用平板电脑听力计对接受高剂量 IV 氨基糖苷类药物治疗的小儿囊性纤维化患者进行常规听力筛查的成本效益分析。

Cost-effectiveness of implementing routine hearing screening using a tablet audiometer for pediatric cystic fibrosis patients receiving high-dose IV aminoglycosides.

机构信息

Division of Pharmaceutical Outcomes and Policy, University of North Carolina at Chapel Hill.

University of North Carolina Medical Center, Chapel Hill.

出版信息

J Manag Care Spec Pharm. 2021 Feb;27(2):157-165. doi: 10.18553/jmcp.2021.27.2.157.

Abstract

Cystic fibrosis (CF) patients who receive high-dose aminoglycosides can acquire inner ear damage and subsequent hearing loss. There is no current standard protocol for assessing ototoxicity in CF centers in the United States. To evaluate the cost-effectiveness of a pharmacist-implemented routine hearing screening for ototoxicity among pediatric patients using a clinically validated tablet audiometer to allow for earlier detection of hearing loss in an exploratory analysis. A Markov decision-analytic model was developed to assess the cost-effectiveness of implementing routine screening with monthly cycles over a 3-year time horizon. The model measured the difference in promptly detected hearing loss, delayed detected hearing loss, and undetected hearing loss, compared with current screening practices. Model inputs were obtained through a comprehensive literature review. Primary model outcomes included total health care costs and quality-adjusted life-years (QALYs) gained with a 3% yearly discount. One-way, two-way, and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. In a hypothetical cohort of 100 patients, routine screening using a tablet audiometer increased promptly detected hearing loss by 8 patients. There was an incremental gain of 3.2 QALYs at an increased cost of $333,826 compared with current screening practices. This resulted in an incremental cost-effectiveness ratio (ICER) of $103,771 per QALY. In the 1-way sensitivity analysis, the ICER ranged between $64,345 and $258,830 per QALY. Using a tablet audiometer for routine hearing screening appears to be a cost-effective option at a $150,000 per QALY willingness-to-pay threshold when only considering the immediate benefits gained. This analysis did not examine the long-term effects of early detection in language development for pediatric patients. Huang reports funding from the University of North Carolina and GlaxoSmithKline Health Outcomes Fellowship. GlaxoSmithKline had no involvement in the study creation, analysis, or manuscript composition. The other authors have nothing to disclose.

摘要

接受高剂量氨基糖苷类药物治疗的囊性纤维化 (CF) 患者可能会出现内耳损伤和随后的听力损失。目前,美国的 CF 中心没有评估耳毒性的标准方案。为了评估药剂师实施的常规听力筛查对使用经过临床验证的平板电脑听力计进行耳毒性检测的成本效益,以便在探索性分析中更早地发现听力损失。

建立了一个马尔可夫决策分析模型,以评估在 3 年时间内每月进行常规筛查的成本效益。该模型通过综合文献回顾获得模型输入。主要模型结果包括与当前筛查实践相比,及时发现的听力损失、延迟发现的听力损失和未发现的听力损失之间的差异。模型投入通过全面的文献回顾获得。主要模型结果包括与当前筛查实践相比,及时发现的听力损失、延迟发现的听力损失和未发现的听力损失之间的差异。模型输入通过全面的文献回顾获得。主要模型结果包括与当前筛查实践相比,及时发现的听力损失、延迟发现的听力损失和未发现的听力损失之间的差异。模型投入通过全面的文献回顾获得。主要模型结果包括与当前筛查实践相比,及时发现的听力损失、延迟发现的听力损失和未发现的听力损失之间的差异。模型投入通过全面的文献回顾获得。主要模型结果包括总医疗保健成本和获得的质量调整生命年 (QALY),每年贴现率为 3%。进行了单向、双向和概率敏感性分析,以评估模型的不确定性。在一个 100 名患者的假设队列中,使用平板电脑听力计进行常规筛查可使听力损失的及时发现增加 8 例。与当前的筛查实践相比,在增加 333,826 美元成本的情况下,额外获得了 3.2 个 QALYs,这导致增量成本效益比 (ICER) 为每 QALY 103,771 美元。在单向敏感性分析中,ICER 在每 QALY 64,345 美元至 258,830 美元之间。

仅考虑立即获得的收益,使用平板电脑听力计进行常规听力筛查似乎是一种具有成本效益的选择,其愿意支付的每 QALY 为 150,000 美元。该分析并未检查早期检测对儿科患者语言发展的长期影响。

Huang 报告说,他从北卡罗来纳大学和葛兰素史克健康结果奖学金获得了资金。葛兰素史克公司没有参与研究的创建、分析或论文的撰写。其他作者没有什么可披露的。

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本文引用的文献

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Audiometric assessment of pediatric patients with cystic fibrosis.儿童囊性纤维化患者的听力评估。
J Cyst Fibros. 2018 May;17(3):383-390. doi: 10.1016/j.jcf.2017.10.007. Epub 2017 Dec 27.

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