Exploring Heterogeneity in the Cost-Effectiveness of High-Flow Nasal Cannula Therapy in Acutely Ill Children-Insights From the Step-Up First-line Support for Assistance in Breathing in Children Trial Using a Machine Learning Method.

IF 4.9 2区 医学 Q1 ECONOMICS Value in Health Pub Date : 2024-09-28 DOI:10.1016/j.jval.2024.08.008
Zaid Hattab, Silvia Moler-Zapata, Edel Doherty, Zia Sadique, Padmanabhan Ramnarayan, Stephen O'Neill
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Abstract

Objectives: To investigate heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support.

Methods: Using data from the First-line Support for Assistance in Breathing in Children trial, we explore heterogeneity at the patient and subgroup levels using 2 causal forest approaches and a seemingly unrelated regression approach for comparison. First-line Support for Assistance in Breathing in Children is a noninferiority randomized controlled trial (ISRCTN60048867) involving 24 UK pediatric intensive care units. The Step-up trial focuses on acutely ill children aged 0 to 15 years, requiring noninvasive respiratory support. A total of 600 children were randomly assigned to HFNC and CPAP groups in a 1:1 allocation ratio, with 94 patients excluded because of data unavailability.

Results: The primary outcome is the incremental net monetary benefit (INB) of HFNC compared with CPAP, using a willingness-to-pay threshold of £20 000 per quality-adjusted life year gain. INB is derived from total costs and quality-adjusted life years at 6 months. Subgroup analysis showed that some subgroups, such as male children, those aged less than 12 months, and those without severe respiratory distress at randomization, had more favorable INB results. Patient-level analysis revealed heterogeneity in INB estimates, particularly driven by the cost component, with greater uncertainty for those with higher INBs.

Conclusions: The estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous, which highlights the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.

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探索急性病儿童高流量鼻插管 (HFNC) 治疗的成本效益异质性--利用机器学习方法从 FIRST-ABC 高级试验中获得的启示。
目的研究在需要无创呼吸支持的急性期患儿中,高流量鼻插管(HFNC)疗法与持续气道正压(CPAP)疗法成本效益的异质性:利用儿童呼吸辅助系统第一线支持(FIRST-ABC)试验的数据,我们使用两种因果森林方法和一种看似不相关的回归(SUR)方法在患者和亚组水平上探讨了异质性。FIRST-ABC 是一项非劣效性随机对照试验(ISRCTN60048867),涉及 24 个英国儿科重症监护病房。该 "升级 "试验主要针对需要无创呼吸支持的 0 至 15 岁急症儿童。共有 600 名儿童按 1:1 的分配比例被随机分配到 HFNC 组和 CPAP 组,其中 94 名患者因无法获得数据而被排除:主要结果是 HFNC 与 CPAP 相比的增量净货币收益 (INB),使用的支付意愿阈值为每 QALY 收益 20,000 英镑。INB 由总成本和 6 个月的质量调整生命年 (QALY) 得出。亚组分析表明,一些亚组,如男性儿童、年龄小于 12 个月的儿童和随机时无严重呼吸困难的儿童,INB 结果更有利。患者层面的分析显示,INB估计值存在异质性,尤其是在成本部分,INB较高者的不确定性更大:结论:对于特定的患者亚群,HFNC 的总体 INB 估计值要大得多,这表明 HFNC 的成本效益可能存在异质性,突出了在评估 HFNC 成本效益时考虑患者特征的重要性。
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来源期刊
Value in Health
Value in Health 医学-卫生保健
CiteScore
6.90
自引率
6.70%
发文量
3064
审稿时长
3-8 weeks
期刊介绍: Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
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