Exploring cost-effectiveness heterogeneity of high flow nasal cannula (HFNC) therapy in acutely ill children - Insights from step-up FIRST-ABC trial using a machine learning method.

To investigate heterogeneity in the cost-effectiveness of high flow nasal cannula (HFNC) therapy compared to continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support. Using data from the FIRST-line support for Assistance in Breathing in Children (FIRST-ABC) trial, we explore heterogeneity at the patient and subgroup levels using two causal forest approaches and a seemingly unrelated regression (SUR) approach for comparison. FIRST-ABC is a non-inferiority randomized controlled trial (ISRCTN60048867) involving 24 UK paediatric intensive care units. The 'step-up' trial focuses on acutely ill children aged 0 to 15 years, requiring non-invasive 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 due to data unavailability. The primary outcome is the incremental net monetary benefit (INB) of HFNC compared to CPAP, using a willingness-to-pay threshold of £20,000 per QALY gain. INB is derived from total costs and Quality Adjusted Life Years (QALYs) at six 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. The estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous which highlight the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.

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