Table 1 Outcomes and analysis models
Outcome | Endpoints | Category | Analysis model |
|---|---|---|---|
Primary | |||
Objective SCORAD | Difference in the objective SCORAD in both groups after 24 weeks of treatment | Continuous outcome measures | Mixed-effects linear regressions (primary) Instrumental variable regression |
Secondary | |||
Treatment failure | Participants who have persistent severe eczema despite 2 courses of rescue therapy (0.5 to 1 mg/kg/day of orally administered prednisolone for a week at a maximum dose of 40 mg/day, followed by a week at 50% of this dose) | Binary outcome measures | Logistic regression model |
Alternative systemic therapy | Requirement for alternative systemic therapy | Binary outcome measures | Logistic regression model |
Eczema quality of life | • POEM • (C)DLQI | Continuous outcome measures | ANCOVA |
Eczema severity | • Subjective SCORAD • EASI score | Continuous outcome measures | ANCOVA |
Effect on co-existing allergic disease | PADQLQ | Continuous outcome measures | ANCOVA |
Number of eczema exacerbationsc | • Clinician-diagnosed exacerbation of eczema or • Increase on SCORAD by 15 points from last recorded SCORAD with participant/parent perception of worsening eczema | Numerical outcome measures | Poisson regression Negative binomial regression model Zero-inflated Poisson regression model (as appropriate) |
Infective episodes of eczemac | Clinician-diagnosed and -treated infective episode of eczema, or clinically apparent, culture-positive infective exacerbations | Numerical outcome measures | Poisson regression Negative binomial regression model Zero-inflated Poisson regression model(as appropriate) |
Allergen-specific IgEa | Change in allergen-specific IgE | Continuous outcome measures | ANCOVA |
Reactivity to food and aeroallergensa | Change in skin-prick test reactivity to food and aeroallergens | Numerical outcome measures | Poisson regression Negative binomial regression model Zero-inflated Poisson regression model |
Safety | |||
Adverse eventsb | Spontaneously reported AE will be collected throughout the follow-up period | Binary outcome | Descriptive analysis |
Urea and electrolytes, creatinine, FBC, eosinophils, LFT, IgE, vitamin D, iron level, bone profile | Surveillance tests where abnormal ranges are defined using the ranges specified by the processing laboratory | Binary outcome | Descriptive analysis |