Related Experiment Video
Updated: Dec 3, 2025
Breath Collection from Children for Disease Biomarker Discovery
Published on: February 14, 2019
First-line support for assistance in breathing in children: statistical and health economic analysis plan for the
Izabella Orzechowska1, M Zia Sadique2, Karen Thomas3
1Clinical Trials Unit, Intensive Care National Audit & Research Centre (ICNARC), Napier House, 24 High Holborn, London, WC1V 6AZ, UK. izabella.orzechowska@icnarc.org.
Insights
The FIRST-ABC trial compares high flow nasal cannula (HFNC) to continuous positive airway pressure (CPAP) for respiratory support in critically ill children. This study details the statistical and economic analysis plan to ensure unbiased results.
Area of Science:
- Pediatric critical care medicine
- Respiratory support interventions
- Clinical trial methodology
Background:
- Two pragmatic, multicenter, non-inferiority randomized clinical trials (FIRST-ABC) evaluate high flow nasal cannula (HFNC) versus continuous positive airway pressure (CPAP).
- These trials focus on critically ill children requiring non-invasive respiratory support (NRS).
- Trial registration: ISRCTN60048867.
Purpose of the Study:
- To outline the pre-specified statistical and health economic analysis plan for the FIRST-ABC trial.
- To minimize analytical bias by defining analyses before patient recruitment and data collection completion.
- To ensure robust evaluation of non-inferiority between HFNC and CPAP.
Main Methods:
- The statistical analysis plan was developed by chief investigators and statisticians.
- Primary outcome: time to liberation from respiratory support; sensitivity analysis using time to start weaning of NRS.
- Health economic analysis includes costs, quality-adjusted life years (QALYs), and cost-effectiveness up to 6 months.
Main Results:
- Primary effect estimate: adjusted hazard ratio with 95% confidence interval.
- Subgroup analyses will investigate treatment interactions with baseline covariates.
- Analyses will be conducted separately for each of the two trials.
Conclusions:
- The FIRST-ABC trial assesses the non-inferiority of HFNC versus CPAP in critically ill children.
- A pre-specified statistical and health economics analysis plan was established before trial completion.
- This approach aims to minimize analytical bias in the study findings.
Background:
The FIRST-ABC trial comprises of two pragmatic, multicentre, parallel groups, non-inferiority randomised clinical trials designed to evaluate the clinical non-inferiority of first-line use of high flow nasal cannula (HFNC) to continuous positive airway pressure (CPAP) in critically ill children who require non-invasive respiratory support (NRS).
Objectives:
To describe the pre-specified statistical and health economic analysis for the FIRST-ABC trial before completion of patient recruitment and data collection.
Methods:
The statistical analysis plan was designed by the chief investigators and statisticians. We define the primary and secondary outcomes, summarise methods for data collection and safety monitoring, and present a detailed description of the planned statistical and health economic analysis.
Results:
The primary clinical outcome is time to liberation from respiratory support. The primary effect estimate will be the adjusted hazard ratio, reported with a 95% confidence interval. As a sensitivity analysis, the primary analysis will be repeated using time to start weaning of NRS. Subgroup analyses will be performed to test for interactions between the effect of allocated treatment group and pre-specified baseline covariates. The health economic analysis will follow the intention-to-treat principle and report the mean (95% confidence interval) incremental costs, quality-adjusted life years (QALYs) and cost-effectiveness up to 6 months. All analyses will be performed separately for each of the two trials, and any results will not be combined.
Conclusion:
The FIRST-ABC trial will assess the non-inferiority of HFNC compared to CPAP in two parallel trials with shared infrastructure (step-up RCT and step-down RCT). We have developed a pre-specified statistical and health economics analysis plan for the FIRST-ABC study before trial completion to minimise analytical bias.
Trial Registration:
ISRCTN ISRCTN60048867 . Registered on 19 June 2019.
Related Concept Videos
01:23Assessment of Respiration
Subjective Assessment: Nurses interview the patient to gather information directly during the subjective assessment. It includes questions about the individual's medical history, medications, and symptoms, focusing on past respiratory conditions like...
01:19Respiratory Assessment: Purpose and Indications
Objectives and Importance:
The primary goal of respiratory assessment is to evaluate patients at early risk of clinical deterioration. Since respiratory distress often precedes other signs of declining health, breathing patterns and sounds become a...
01:30Asthma-IV: Nursing Management
First, in...
01:29Acute Respiratory Failure-V
Ensure that patients are monitored continuously for their response to therapy, including changes in...
01:27Chronic Obstructive Pulmonary Disease-IV: Assessement and Diagnostic Studies
Medical History
01:28Physical Assessment of the Respiratory Tract I: Health History
Subjective Data
Subjective data provides vital information about the patient's health history and symptoms. This data is typically collected through interviews in which patients describe their experiences, symptoms, and concerns.
Health history and...