Comparative Effectiveness of Extra-Fine Particle Inhaled Corticosteroid (ICS) and Alternative Guideline-Recommended Step-Up Options in Pre-School Children

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Observational, matched cohort study drawing on retrospective, EMRs from the OPCRD

Study drug and medical condition

• Medical condition to be studied:
  Asthma

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Children (2 to < 12 years)
• Estimated number of subjects:
  11000

Study design details

• Main study objective:
  The aim of this study is to test the hypothesis that use of extra-fine ICS in pre-school children (i.e. ≤5 years of age) with asthma/wheeze will achieve better outcomes than treatment alternatives (i.e. non extra-fine ICS, LTRA, or SABA).
• Outcomes:
  Exacerbations (ATS/ERS definition) defined as occurrence of an: • Asthma-related: Hospital admissions OR A&E attendance, OR• An acute course of oral steroids (coded for asthma or wheeze). -Acute respiratory event-Risk Domain Asthma Control -Overall Asthma Control (OAC)-Treatment stability-SABA usage-Controller-to-Reliever Ratio-Oral Thrush
• Data analysis plan:
  Statistically significant results will be defined as p<0.05 and trends as 0.05≤p<0.10. Summary statistics will be presented as appropriate for each variable: • Variables measured on the interval or ratio scale: n and % of non-missing data, mean (standard deviation) and median (inter-quartile range)• Categorical variables: n (%) of non-missing data, n (%) per categoryTreatment cohorts were compared at baseline using the Mann–Whitney test for continuous variables and the Chi-squared test for categorical variables.Conditional logistic regression will be used to compare baseline characteristics between matched cohorts. Any variables that remained potentially different between matched cohorts at baseline (p<0.10) will be included as potential confounding factors in the outcome analysis. Conditional logistic regression will be used to compare cohorts for binary outcomes, and a conditional Poisson regression model will be used to compare outcome exacerbation rates.