title: Efficacy and safety of oral budesonide suspension in pediatric patients with eosinophilic esophagitis.
abstract: OBJECTIVE
  No treatment has been approved by the U.S. Food and Drug Administration for eosinophilic esophagitis (EoE). We investigated the efficacy and safety of a new formulation of oral budesonide suspension (OBS), a corticosteroid, in a prospective, placebo-controlled, dose-ranging study.
  
  
  METHODS
  Subjects 2-18 years old with symptoms of EoE and peak eosinophil counts ≥20/high-power field at ≥2 levels of the esophagus were randomly assigned to groups given placebo or low-dose, medium-dose, or high-dose OBS for 12 weeks. Doses and volumes were adjusted on the basis of patients' age to cover the entire esophagus. The primary efficacy end point was compound response to therapy (peak eosinophil counts ≤6/high-power field at all levels of the esophagus and ≥50% reduction in EoE symptom score). Multiple safety parameters were evaluated.
  
  
  RESULTS
  Data from 71 subjects who completed all efficacy assessments were included in the primary efficacy analysis. At the end of 12 weeks, there were significantly greater percentages of responders in groups given medium-dose OBS (52.6%, P = .0092) and high-dose OBS (47.1%, P = .0174) than in the group given placebo (5.6%); there was no significant difference in percentages of responders between the low-dose OBS (11.8%) and placebo groups (P = .5282). The significant compound responses noted in the medium-dose and high-dose OBS groups were accounted for by the significant histologic responses; in contrast, all 4 groups (including the placebo group) had large symptom responses, and there was no significant difference in the percentage of subjects with a symptom response in either OBS group compared with the placebo group (P ≥ .1235). There were no unexpected safety concerns or signals.
  
  
  CONCLUSIONS
  Peak eosinophil counts were significantly reduced throughout the esophagus in pediatric patients with EoE who were given medium-dose and high-dose OBS. There was a large symptom response to placebo that was similar to symptom responses in the OBS groups; symptom response did not distinguish OBS from placebo. ClinicalTrials.gov number, NCT00762073.
authors.affiliation: Section of Pediatric Gastroenterology, Hepatology and Nutrition, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana. Electronic address: sgupta@iupui.edu.
authors.forename: Sandeep K
authors.initials: SK
authors.lastname: Gupta
authors.affiliation: Meritage Pharma, Inc, San Diego, California.
authors.forename: Joanne M
authors.initials: JM
authors.lastname: Vitanza
authors.affiliation: Department of Pathology and Laboratory Medicine, University of Cincinnati, Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Margaret H
authors.initials: MH
authors.lastname: Collins
mesh_terms: D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D019819:Budesonide / Q000009:adverse effects* / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D064420:Drug-Related Side Effects and Adverse Reactions / Q000453:epidemiology; D057765:Eosinophilic Esophagitis / Q000188:drug therapy*; D004804:Eosinophils / Q000276:immunology; D004947:Esophagus / Q000473:pathology; D005260:Female; D006651:Histocytochemistry; D006801:Humans; D007958:Leukocyte Count; D008297:Male; D013535:Suspensions / Q000009:adverse effects* / Q000627:therapeutic use*; D016896:Treatment Outcome; D014481:United States
chemical_list: D000893:Anti-Inflammatory Agents; D013535:Suspensions; D019819:Budesonide
keywords: Clinical Trial; Esophageal Eosinophil; Symptom Score; Topical Corticosteroid