Health Economics, Department of Health Services Research and Policy, Public Health and Policy Faculty, London School of Hygiene and Tropical Medicine, London, UK.
Clinical Trials Unit, Intensive Care National Audit & Research Centre, London, UK.
Crit Care. 2024 Nov 25;28(1):386. doi: 10.1186/s13054-024-05148-y.
High flow nasal cannula therapy (HFNC) and continuous positive airway pressure (CPAP) are two widely used modes of non-invasive respiratory support in paediatric critical care units. The FIRST-ABC randomised controlled trials (RCTs) evaluated the clinical and cost-effectiveness of HFNC compared with CPAP in two distinct critical care populations: acutely ill children ('step-up' RCT) and extubated children ('step-down' RCT). Clinical effectiveness findings (time to liberation from all forms of respiratory support) showed that HFNC was non-inferior to CPAP in the step-up RCT, but failed to meet non-inferiority criteria in the step-down RCT. This study evaluates the cost-effectiveness of HFNC versus CPAP.
All-cause mortality, health-related Quality of Life (HrQoL), and costs up to six months were reported using FIRST-ABC RCTs data. HrQoL was measured with the age-appropriate Paediatric Quality of Life Generic Core Scales questionnaire and mapped onto the Child Health Utility 9D index score at six months. Quality-Adjusted Life Years (QALYs) were estimated by combining HrQoL with mortality. Costs at six months were calculated by measuring and valuing healthcare resources used in paediatric critical care units, general medical wards and wider health service. The cost-effectiveness analysis used regression methods to report the cost-effectiveness of HFNC versus CPAP at six months and summarised the uncertainties around the incremental cost-effectiveness results.
In both RCTs, the incremental QALYs at six months were similar between the randomised groups. The estimated incremental cost at six months was - £4565 (95% CI - £11,499 to £2368) and - £5702 (95% CI - £11,328 to - £75) for step-down and step-up RCT, respectively. The incremental net benefits of HFNC versus CPAP in step-down RCT and step-up RCT were £4388 (95% CI - £2551 to £11,327) and £5628 (95% CI - £8 to £11,264) respectively. The cost-effectiveness results were surrounded by considerable uncertainties. The results were similar across most pre-specified subgroups, and the base case results were robust to alternative assumptions.
HFNC compared to CPAP as non-invasive respiratory support for critically-ill children in paediatric critical care units reduces mean costs and is relatively cost-effective overall and for key subgroups, although there is considerable statistical uncertainty surrounding this result.
高流量鼻导管治疗（HFNC）和持续气道正压通气（CPAP）是儿科重症监护病房中两种广泛使用的非侵入性呼吸支持模式。FIRST-ABC 随机对照试验（RCT）评估了 HFNC 与 CPAP 在两个不同重症监护人群中的临床和成本效益：急性疾病患儿（“逐步升级” RCT）和拔管患儿（“逐步降低” RCT）。临床有效性发现（从所有形式的呼吸支持中解放出来的时间）表明，HFNC 在逐步升级 RCT 中不比 CPAP 差，但在逐步降低 RCT 中未能达到非劣效性标准。本研究评估了 HFNC 与 CPAP 的成本效益。
使用 FIRST-ABC RCT 数据报告全因死亡率、健康相关生活质量（HrQoL）和六个月内的成本。HrQoL 使用适合年龄的儿科生活质量通用核心量表问卷进行测量，并在六个月时映射到儿童健康效用 9D 指数评分上。通过将 HrQoL 与死亡率相结合，估计了质量调整生命年（QALYs）。六个月的成本是通过测量和评估儿科重症监护病房、普通医疗病房和更广泛的卫生服务中使用的医疗资源来计算的。成本效益分析使用回归方法报告了六个月时 HFNC 与 CPAP 的成本效益，并总结了增量成本效益结果的不确定性。
在两项 RCT 中，随机分组在六个月时的增量 QALYs 相似。六个月的估计增量成本分别为 - £4565（95%CI - £11,499 至 £2368）和 - £5702（95%CI - £11,328 至 £75），用于逐步降低和逐步升级 RCT。在逐步降低 RCT 和逐步升级 RCT 中，HFNC 与 CPAP 的增量净效益分别为 £4388（95%CI - £2551 至 £11,327）和 £5628（95%CI - £8 至 £11,264）。成本效益结果存在相当大的不确定性。在大多数预设亚组中，结果相似，基础病例结果对替代假设具有稳健性。
HFNC 与 CPAP 相比，作为儿科重症监护病房中危重病儿的非侵入性呼吸支持，可降低平均成本，总体上和关键亚组中相对具有成本效益，尽管这一结果存在相当大的统计不确定性。
