Skip to main content

Advertisement

Table 1 Outcomes and analysis models

From: The role of anti-IgE (omalizumab/Xolair) in the management of severe recalcitrant paediatric atopic eczema (ADAPT): statistical analysis plan

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
  1. aOnly collected at screening and 24 weeks of treatment. The remaining outcomes are collected at baseline, 4-weekly during the 24 weeks of treatment, 36 weeks and 48 weeks
  2. bBlood test and urine samples will be collected at baseline, 24 weeks, 36 weeks and 48 weeks. Clinical observations will be examined at every visit
  3. cChi‐square goodness‐of‐fit tests will be used to select the suitable model
  4. ANCOVA analysis of covariance, IgE immunoglobulin E, SCORAD SCORing Atopic Dermatitis, PADQLQ Paediatric Allergic Disease Quality of Life Questionnaire, (C)DLQI (Children’s) Dermatology Life Quality Index, EASI Eczema Area and Severity Index, AE adverse events, POEM Patient-oriented Eczema Measure, FBC full blood count, LFT liver function test