Asthma is the most common long-term disease in children in the UK. It causes cough and difficulty
breathing. These symptoms get worse during asthma attacks. The main treatment is a preventer inhaler, containing steroids.
We are going to do a trial to help us learn how best to treat children with mild asthma. We want to find out if taking a preventer inhaler only on days when a child has symptoms is as effective as taking it every day. Less preventer inhaler may make their asthma symptoms worse, but using them too much can cause unwanted side-effects. The study will involve around 2000 children from GP practices across the UK. Parents of children aged 6-15 with mild asthma at these practices will be asked if they would like to take part. If the parents agree, their child will be randomised (assigned by chance) either to take their preventer inhaler every day, or only when they have symptoms of asthma. We want to know if this increases the chances of having an asthma attack that needs treatment. We also want to see whether there is a difference between the two approaches in terms of asthma symptoms, hospital admissions, the child’s overall quality of life, and overall costs (for example, costs for prescriptions, GP appointments and hospital visits).
This study will answer important questions on how best to treat children with mild asthma. This will benefit the wider public by helping to guide clinical practice on treatment of mild asthma in children.
Aims: To evaluate whether in children (6 to 16 years old) with mild asthma, symptom-driven use of inhaled corticosteroid (ICS) is non-inferior to maintenance ICS with regards to the risk of asthma attacks requiring oral corticosteroids (OCS).
Design: Pragmatic open-label Randomised Controlled Trial (RCT), in general practices across the UK who provide data to CPRD.
Outcomes: Collected from the electronic health record (EHR): Hospital Episode Statistics (HES) admission data (to determine hospitalisations); HES A&E data (SAEs, asthma exacerbations and treatment failure involving ICS), ONS (mortality). Patient-reported outcomes: collected on IRSP and REDCap (an electronic data capture platform). All data will be processed and provided by CPRD and will be re-pseudonymised using a study specific primary key.
Interventions: Children, already prescribed ICS, will be randomised 1:1 to either continue as prescribed with maintenance ICS use every day (control arm) or reduce to symptom-driven ICS use (intervention arm) which entails using the ICS only on days when Short-acting beta-2 agonists (SABAs) are required. The treatment period is 12 months.
Analysis: 1854 children (927 per group) will provide 80% power with a one-sided 2.5% significance level to reject the null hypothesis that the symptom-driven regime is inferior to the maintenance regime. The primary outcome, and other binary outcomes, will be analysed using logistic regression. Quality of life scores will be analysed using analysis of covariance adjusted for baseline score Health economic analyses will estimate the cost-effectiveness (incremental cost per quality-adjusted life year, QALY gained) of symptom-driven use of ICS.
Public Health Benefit: This study will answer important questions on how best to treat children with mild asthma which may benefit patients through reduced exposure to inhaled steroids as well as determining the most cost-effective treatments. This will support the reconsideration of national and international guidance on treatment for children with asthma.
• Risk of asthma attack requiring oral corticosteroid (OCS) in the study population
• The cost-effectiveness (incremental cost per quality-adjusted life year, QALY gained) of symptom-driven use of ICS - numbers of prescriptions and interactions with healthcare providers
• Time to first asthma attack requiring treatment with OCS
• Asthma control
• Unscheduled healthcare utilisation
• All cause hospitalisation
• Health Related Quality of Life
• Treatment failure
• Cumulative dose of ICS over the 12 month treatment period
• All cause mortality
Ian Sinha - Chief Investigator - University of Liverpool
Susanna Dodd - Corresponding Applicant - University of Liverpool
Christiane Marin - Collaborator - CPRD
Clare Iles - Collaborator - CPRD
Dyfrig Hughes - Collaborator - Bangor University
Giovanna Culeddu - Collaborator - Bangor University
Paula Williamson - Collaborator - University of Liverpool
Rebecca Ghosh - Collaborator - CPRD
Susan Beatty - Collaborator - CPRD
HES Accident and Emergency;HES Admitted Patient Care;ONS Death Registration Data;Patient Level Carstairs Index