PRESTO试验的成本效果分析：一项评估头颈癌患者放（化）疗期间预防性吞咽练习的三臂随机对照试验

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中文摘要

背景： 对口咽部头颈癌（HNC）患者的研究表明，在放（化）疗期间进行预防性吞咽练习（PSE）可对肌肉力量、吞咽功能和生活质量（QOL）产生积极影响。PRESTO试验的主要分析显示，与使用练习日记或数字应用程序的自我指导练习相比，每日由言语语言病理学家（SLP）指导练习时PSE的依从率更高。此外，更高的依从率与吞咽功能和生活质量更大的改善相关。然而，鉴于医疗资源有限，成本效果分析对于指导此类康复策略在吞咽障碍照护中的实施至关重要。这项PRESTO数据的二次分析比较了三种不同服务模式的成本和健康效应：基于应用程序、治疗师主导（每日面对面，放（化）疗期间20次）和纸质指导治疗。
方法： 150例因口咽部HNC接受放（化）疗（C）RT的患者被随机分配到三个练习组之一。所有患者均执行相同的PSE方案。治疗方案要求在（C）RT治疗程序的前4周内每日训练。治疗组患者因此共接受20次面对面治疗。健康结局以基于EQ5D5L工具的效用值表示，在基线、放（化）疗后和放（化）疗后3个月测量。成本从卫生服务视角估算，包括员工时间、治疗资源和消耗品。增量成本效果比（ICER）按Δ成本/ΔQALY计算。
结果： 各组间效用值未发现显著差异。仅观察到显著的时间效应，效用值从基线到放（化）疗后显著下降，并从放（化）疗后到放（化）疗后3个月显著上升。治疗师主导干预与最高成本相关。应用程序组干预在成本效果方面被纸质组所支配，因为效用值相当但应用程序组成本更高。因此，ICER在纸质组与治疗师组之间计算，结果为124 061.54欧元/QALY。
结论： 尽管3个月后总体健康相关生活质量效应为正向，但每日由治疗师主导实施PSE治疗相对于纸质自我管理并不具有成本效果。在当前资源约束下，低成本实施模式代表最具经济效率的选择。未来研究应探索混合式或针对性治疗师参与是否能通过保留临床获益同时降低成本来产生更好的价值。

英文摘要

BACKGROUND: Research on oropharyngeal head and neck cancer (HNC) patients indicates that prophylactic swallowing exercises (PSE) during (C)RT can positively affect muscle strength, swallowing function and quality of life (QOL). Primary analyses of the PRESTO-trial showed higher adherence rates for PSE when exercising daily with a speech-language pathologist (SLP), compared to self-guided exercises with either an exercise diary or a digital application. Additionally, higher adherence rates were related to greater improvements in swallowing function and QOL. However, given limited healthcare resources, cost-effectiveness analyses are essential to inform the implementation of such rehabilitation strategies in dysphagia care. This secondary analysis of the PRESTO-data compares the costs and health effects of the three different service models: app-based, therapist-led (daily face-to-face, 20 sessions during (C)RT), and paper-led therapy.
METHODS: One hundred fifty patients receiving (chemo)radiotherapy (C)RT for oropharyngeal HNC were randomly assigned to one of the three exercise groups. All performing the same PSE protocol. The treatment protocol required daily training during the first 4 weeks of the (C)RT treatment program. Patients in the therapy group thereby received a total of 20 face-to-face therapy sessions. Health outcomes were expressed as utilities based on the EQ5D5L instrument measured at baseline, post (C)RT and 3 months post (C)RT. Costs were estimated from a health service perspective, including staff time, therapy resources and consumables. Incremental cost-effectiveness ratios (ICERs) were calculated as ΔCosts/ ΔQALYs.
RESULTS: No significant between-group differences were found for utilities. Only significant time effects were observed, with a significant decrease in utilities from baseline to post (C)RT and a significant increase from post (C)RT to 3 months post (C)RT. The therapist-led intervention was associated with the highest costs. The app-group intervention was dominated by the paper group in terms of cost-effectiveness as the utilities were comparable but at a higher cost in the app-group. Consequently, the ICER was computed between the paper-group and therapist-group and resulted in € 124 061,54/QALY.
CONCLUSIONS: Despite the overall positive HR-QOL effects post 3 months, the daily therapist-led delivery of PSE therapy was not cost-effective relative to paper-based self-management. Under current resource constraints, low-cost delivery models represent the most economically efficient option. Future research should explore whether hybrid or targeted therapist involvement yields better value by preserving clinical benefit while reducing cost.