INDIRECT TREATMENT COMPARISON OF BUDESONIDE ORAL SUSPENSION VS DUPILUMAB IN PAEDIATRIC EOE
Author(s)
Andrea Latour, MSc1, Kacper Swierk, MBiol2, Charlie Hewitt, MSc3, Wolfram Hildebrandt, MSc4.
1Remap Consulting GmbH, Zug, Switzerland, 2Remap Consulting UK Ltd, Cheshire, United Kingdom, 3Remap Consulting, CHESHIRE, United Kingdom, 4Dr. Falk Pharma GmbH, Freiburg, Germany.
1Remap Consulting GmbH, Zug, Switzerland, 2Remap Consulting UK Ltd, Cheshire, United Kingdom, 3Remap Consulting, CHESHIRE, United Kingdom, 4Dr. Falk Pharma GmbH, Freiburg, Germany.
OBJECTIVES: Budesonide oral suspension (BOS) and dupilumab demonstrated significant efficacy versus placebo in paediatric eosinophilic oesophagitis (EoE), however, no head-to-head studies comparing BOS with dupilumab are available. This study compared the efficacy of BOS versus dupilumab using an indirect treatment comparison (ITC).
METHODS: A Bucher ITC was conducted using placebo as the common comparator. Efficacy data for BOS were derived from PEDEOS-1 [EudraCT No: 2017-003737-29], while dupilumab data were obtained from the EoE KIDS trial in children aged 1-11 years [EudraCT No: 2019-003078-24] and LIBERTY EoE TREET in adolescents aged 12-17 years [EudraCT No: 2018-000844-25]. The primary endpoint was histologic remission, defined as ~<5 eosinophils/high-power field (eos/hpf, transformed from eos/mm2) for budesonide’s studies and ≤6 for dupilumab’s studies and, assessed separately by age and dosing subgroup. The key trial characteristics and eligibility criteria were considered sufficiently comparable to support the transitivity assumption underlying the anchored Bucher ITC. Due to sparse event data and zero-event placebo arms, analyses included odds ratios (ORs) using standard Bucher methodology (base case), Haldane-Anscombe continuity corrections, and Firth penalised logistic regression sensitivity analyses.
RESULTS: Across paediatric age and dosing subgroups, BOS demonstrated efficacy numerically equivalent to or greater than dupilumab for histologic remission. In both age subgroups, ITC results numerically favoured BOS versus dupilumab across analyses (base case [combined dose groups] OR of 1.15 in the age 2-11 and 4.69 in the age 12-17 populations, respectively), although confidence intervals were wide due to the limited sample sizes. Sensitivity analyses consistently showed numerically favourable results for BOS compared with dupilumab.
CONCLUSIONS: These findings suggest that BOS provides numerically equivalent or greater histological efficacy compared with dupilumab in children and adolescent patients with EoE. This highlights the clinical benefit of BOS in a population with a high unmet medical need.
METHODS: A Bucher ITC was conducted using placebo as the common comparator. Efficacy data for BOS were derived from PEDEOS-1 [EudraCT No: 2017-003737-29], while dupilumab data were obtained from the EoE KIDS trial in children aged 1-11 years [EudraCT No: 2019-003078-24] and LIBERTY EoE TREET in adolescents aged 12-17 years [EudraCT No: 2018-000844-25]. The primary endpoint was histologic remission, defined as ~<5 eosinophils/high-power field (eos/hpf, transformed from eos/mm2) for budesonide’s studies and ≤6 for dupilumab’s studies and, assessed separately by age and dosing subgroup. The key trial characteristics and eligibility criteria were considered sufficiently comparable to support the transitivity assumption underlying the anchored Bucher ITC. Due to sparse event data and zero-event placebo arms, analyses included odds ratios (ORs) using standard Bucher methodology (base case), Haldane-Anscombe continuity corrections, and Firth penalised logistic regression sensitivity analyses.
RESULTS: Across paediatric age and dosing subgroups, BOS demonstrated efficacy numerically equivalent to or greater than dupilumab for histologic remission. In both age subgroups, ITC results numerically favoured BOS versus dupilumab across analyses (base case [combined dose groups] OR of 1.15 in the age 2-11 and 4.69 in the age 12-17 populations, respectively), although confidence intervals were wide due to the limited sample sizes. Sensitivity analyses consistently showed numerically favourable results for BOS compared with dupilumab.
CONCLUSIONS: These findings suggest that BOS provides numerically equivalent or greater histological efficacy compared with dupilumab in children and adolescent patients with EoE. This highlights the clinical benefit of BOS in a population with a high unmet medical need.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO54
Topic
Clinical Outcomes
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
Gastrointestinal Disorders, Rare & Orphan Diseases