Budesonide oral suspension significantly improves eosinophilic esophagitis histology scoring system results: Analyses from a 12-week, phase 2, randomized, placebo-controlled trial

Margaret H. Collins, Evan S. Dellon, David A Katzka, Ikuo Hirano, James Williams, Lan Lan

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1 Citation (Scopus)

Abstract

Budesonide oral suspension (BOS) is a novel topical corticosteroid, which has been shown to improve symptoms and endoscopic appearance, and reduce peak eosinophil counts in patients with eosinophilic esophagitis (EoE). This trial evaluated the effect of BOS or placebo on the severity (grade) and extent (stage) of 8 histopathologic features observed in EoE, using the validated eosinophilic esophagitis histologic scoring system (EoE HSS). Patients with EoE aged 11 to 40 years with dysphagia were randomized to receive either BOS (2.0 mg twice daily) or placebo for 12 weeks. Mean (SD) EoE HSS grade and stage total scores at baseline for placebo and BOS groups were: Grade, 0.42 (0.16) and 0.49 (0.14), respectively; stage: 0.38 (0.14) and 0.46 (0.11), respectively. These scores significantly decreased (improved) from baseline for patients receiving BOS versus placebo (grade: Least squares mean change [SE]: Placebo vs. BOS,-0.04 [0.03] vs.-0.24 [0.02]; P0.0001; stage:-0.01 [0.02] vs.-0.19 [0.02]; P0.0001). EoE HSS total scores improved for 6 of the 8 and 5 of the 8 histopathologic features for grade and stage, respectively, versus placebo. Change in EoE HSS total scores correlated moderately but significantly with change in endoscopic severity (endoscopic reference score; grade: R=0.5349; stage: R=0.5416; both P0.0001). Change in EoE HSS stage total score correlated weakly with change in Dysphagia Symptom Questionnaire scores (grade: R=0.1925; P=0.0740; stage: R=0.2135; P=0.0471). These data demonstrate that the EoE HSS is a valuable endpoint of treatment response in randomized clinical trials and should be considered for future trials for EoE.

Original languageEnglish (US)
JournalAmerican Journal of Surgical Pathology
DOIs
StateAccepted/In press - Jan 1 2019

Fingerprint

Eosinophilic Esophagitis
Budesonide
Suspensions
Histology
Randomized Controlled Trials
Placebos
Deglutition Disorders
Least-Squares Analysis
Eosinophils
Adrenal Cortex Hormones

Keywords

  • budesonide oral suspension
  • eosinophilic esophagitis
  • histology
  • peak eosinophil count
  • topical corticosteroid

ASJC Scopus subject areas

  • Anatomy
  • Surgery
  • Pathology and Forensic Medicine

Cite this

@article{b45d15cbbaa54afc87d823c636e03633,
title = "Budesonide oral suspension significantly improves eosinophilic esophagitis histology scoring system results: Analyses from a 12-week, phase 2, randomized, placebo-controlled trial",
abstract = "Budesonide oral suspension (BOS) is a novel topical corticosteroid, which has been shown to improve symptoms and endoscopic appearance, and reduce peak eosinophil counts in patients with eosinophilic esophagitis (EoE). This trial evaluated the effect of BOS or placebo on the severity (grade) and extent (stage) of 8 histopathologic features observed in EoE, using the validated eosinophilic esophagitis histologic scoring system (EoE HSS). Patients with EoE aged 11 to 40 years with dysphagia were randomized to receive either BOS (2.0 mg twice daily) or placebo for 12 weeks. Mean (SD) EoE HSS grade and stage total scores at baseline for placebo and BOS groups were: Grade, 0.42 (0.16) and 0.49 (0.14), respectively; stage: 0.38 (0.14) and 0.46 (0.11), respectively. These scores significantly decreased (improved) from baseline for patients receiving BOS versus placebo (grade: Least squares mean change [SE]: Placebo vs. BOS,-0.04 [0.03] vs.-0.24 [0.02]; P0.0001; stage:-0.01 [0.02] vs.-0.19 [0.02]; P0.0001). EoE HSS total scores improved for 6 of the 8 and 5 of the 8 histopathologic features for grade and stage, respectively, versus placebo. Change in EoE HSS total scores correlated moderately but significantly with change in endoscopic severity (endoscopic reference score; grade: R=0.5349; stage: R=0.5416; both P0.0001). Change in EoE HSS stage total score correlated weakly with change in Dysphagia Symptom Questionnaire scores (grade: R=0.1925; P=0.0740; stage: R=0.2135; P=0.0471). These data demonstrate that the EoE HSS is a valuable endpoint of treatment response in randomized clinical trials and should be considered for future trials for EoE.",
keywords = "budesonide oral suspension, eosinophilic esophagitis, histology, peak eosinophil count, topical corticosteroid",
author = "Collins, {Margaret H.} and Dellon, {Evan S.} and Katzka, {David A} and Ikuo Hirano and James Williams and Lan Lan",
year = "2019",
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T1 - Budesonide oral suspension significantly improves eosinophilic esophagitis histology scoring system results

T2 - Analyses from a 12-week, phase 2, randomized, placebo-controlled trial

AU - Collins, Margaret H.

AU - Dellon, Evan S.

AU - Katzka, David A

AU - Hirano, Ikuo

AU - Williams, James

AU - Lan, Lan

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Budesonide oral suspension (BOS) is a novel topical corticosteroid, which has been shown to improve symptoms and endoscopic appearance, and reduce peak eosinophil counts in patients with eosinophilic esophagitis (EoE). This trial evaluated the effect of BOS or placebo on the severity (grade) and extent (stage) of 8 histopathologic features observed in EoE, using the validated eosinophilic esophagitis histologic scoring system (EoE HSS). Patients with EoE aged 11 to 40 years with dysphagia were randomized to receive either BOS (2.0 mg twice daily) or placebo for 12 weeks. Mean (SD) EoE HSS grade and stage total scores at baseline for placebo and BOS groups were: Grade, 0.42 (0.16) and 0.49 (0.14), respectively; stage: 0.38 (0.14) and 0.46 (0.11), respectively. These scores significantly decreased (improved) from baseline for patients receiving BOS versus placebo (grade: Least squares mean change [SE]: Placebo vs. BOS,-0.04 [0.03] vs.-0.24 [0.02]; P0.0001; stage:-0.01 [0.02] vs.-0.19 [0.02]; P0.0001). EoE HSS total scores improved for 6 of the 8 and 5 of the 8 histopathologic features for grade and stage, respectively, versus placebo. Change in EoE HSS total scores correlated moderately but significantly with change in endoscopic severity (endoscopic reference score; grade: R=0.5349; stage: R=0.5416; both P0.0001). Change in EoE HSS stage total score correlated weakly with change in Dysphagia Symptom Questionnaire scores (grade: R=0.1925; P=0.0740; stage: R=0.2135; P=0.0471). These data demonstrate that the EoE HSS is a valuable endpoint of treatment response in randomized clinical trials and should be considered for future trials for EoE.

AB - Budesonide oral suspension (BOS) is a novel topical corticosteroid, which has been shown to improve symptoms and endoscopic appearance, and reduce peak eosinophil counts in patients with eosinophilic esophagitis (EoE). This trial evaluated the effect of BOS or placebo on the severity (grade) and extent (stage) of 8 histopathologic features observed in EoE, using the validated eosinophilic esophagitis histologic scoring system (EoE HSS). Patients with EoE aged 11 to 40 years with dysphagia were randomized to receive either BOS (2.0 mg twice daily) or placebo for 12 weeks. Mean (SD) EoE HSS grade and stage total scores at baseline for placebo and BOS groups were: Grade, 0.42 (0.16) and 0.49 (0.14), respectively; stage: 0.38 (0.14) and 0.46 (0.11), respectively. These scores significantly decreased (improved) from baseline for patients receiving BOS versus placebo (grade: Least squares mean change [SE]: Placebo vs. BOS,-0.04 [0.03] vs.-0.24 [0.02]; P0.0001; stage:-0.01 [0.02] vs.-0.19 [0.02]; P0.0001). EoE HSS total scores improved for 6 of the 8 and 5 of the 8 histopathologic features for grade and stage, respectively, versus placebo. Change in EoE HSS total scores correlated moderately but significantly with change in endoscopic severity (endoscopic reference score; grade: R=0.5349; stage: R=0.5416; both P0.0001). Change in EoE HSS stage total score correlated weakly with change in Dysphagia Symptom Questionnaire scores (grade: R=0.1925; P=0.0740; stage: R=0.2135; P=0.0471). These data demonstrate that the EoE HSS is a valuable endpoint of treatment response in randomized clinical trials and should be considered for future trials for EoE.

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