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.

中文翻译：

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高流量鼻插管治疗与持续气道正压通气在儿科重症监护中无创呼吸支持的成本效益


高流量鼻插管疗法 （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无创呼吸支持模式。FIRST-ABC 随机对照试验 （RCT） 评估了 HFNC 与 CPAP 相比在两个不同的重症监护人群中的临床和成本效益：急症儿童（“升级”RCT）和拔管儿童（“降级”RCT）。临床有效性结果（从各种形式的呼吸支持中解放出来的时间）显示，HFNC 在递增 RCT 中不劣于 CPAP，但在降级 RCT 中未能满足非劣效性标准。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） 和长达 6 个月的成本。使用适合年龄的儿科生活质量通用核心量表问卷测量 HrQoL，并在 6 个月时映射到儿童健康实用 9D 指数评分。通过将 HrQoL 与死亡率相结合来估计质量调整生命年 （QALY）。通过测量和评估儿科重症监护病房、普通内科病房和更广泛的卫生服务中使用的医疗保健资源来计算 6 个月的成本。成本效益分析使用回归方法报告了 HFNC 与 CPAP 在 6 个月时的成本效益，并总结了增量成本效益结果的不确定性。在这两项随机对照试验中，随机分组之间 6 个月时增加的 QALY 相似。降级和升级 RCT 在六个月时的估计增量成本分别为 -4565 英镑（95% CI -11,499 至 2368 英镑）和 -5702 英镑（95% CI -11,328 至 -75 英镑）。 在降级 RCT 和升级 RCT 中，HFNC 与 CPAP 相比的增量净收益分别为 4388 英镑（95% CI − 2551 英镑至 11,327 英镑）和 5628 英镑（95% CI − 8 英镑至 11,264 英镑）。成本效益结果存在相当大的不确定性。大多数预先指定的亚组的结果相似，并且基本情况结果对替代假设是稳健的。与 CPAP 相比，HFNC 作为儿科重症监护病房危重儿童的无创呼吸支持降低了平均成本，并且总体上和关键亚组相对具有成本效益，尽管这一结果存在相当大的统计不确定性。