Oral viscous budesonide is effective in children with eosinophilic esophagitis in a randomized, placebo-controlled trial

Gastroenterology. 2010 Aug;139(2):418-29. doi: 10.1053/j.gastro.2010.05.001. Epub 2010 May 7.

Abstract

Background & aims: Eosinophilic esophagitis (EoE) is caused by immunologic reactions to ingested/inhaled allergens. The diagnosis is considered if >or=15 eosinophils per high-powered field (eos/hpf) are detected in mucosal biopsies. Placebo-controlled studies have not been conducted to evaluate the safety and efficacy of oral viscous budesonide (OVB).

Methods: Children with EoE were randomly assigned to groups that were given OVB (n=15) or placebo (n=9). Patients<5 feet and >or=5 feet tall received 1 mg and 2 mg OVB daily, respectively. All patients received lansoprazole. Duration of treatment was 3 months, followed by repeat endoscopy and biopsies. Patients were classified as responders if their peak eosinophil counts were <or=6 eos/hpf, partial responders were 7-19 eos/hpf, and nonresponders were >or=20 eos/hpf. Baseline and post-treatment symptoms and endoscopic and histologic features were scored.

Results: Thirteen (86.7%) children given OVB (P<.0001) and none who received placebo (P=.3) were classified as responders. Mean pre-/post-treatment peak eosinophil counts were 66.7 and 4.8 eos/hpf, respectively, in the group given OVB (P<.0001); they were 83.9 and 65.6 eos/hpf, respectively, in the group given placebo (P=.3). In the group given OVB, there were significant reductions from baseline values in proximal (P=.002), mid (P=.0003), and distal (P=.001) esophageal eosinophilia. After OVB therapy, compared with baseline, the mean symptom (P=.0007), endoscopy (P=.0005), and histology scores improved (P=.0035) significantly.

Conclusions: OVB is an effective treatment of pan-esophageal disease in children with EoE. OVB improves symptoms and endoscopic and histologic features. Proton pump inhibitor single therapy did not significantly improve esophageal eosinophilia or symptoms of EoE.

Trial registration: ClinicalTrials.gov NCT00638456.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 2-Pyridinylmethylsulfinylbenzimidazoles / therapeutic use
  • Administration, Oral
  • Adolescent
  • Anti-Inflammatory Agents / administration & dosage*
  • Anti-Inflammatory Agents / chemistry
  • Biopsy
  • Budesonide / administration & dosage*
  • Budesonide / chemistry
  • Child
  • Child, Preschool
  • Dosage Forms
  • Double-Blind Method
  • Drug Therapy, Combination
  • Eosinophilia / drug therapy*
  • Eosinophilia / genetics
  • Eosinophilia / pathology
  • Esophagitis / drug therapy*
  • Esophagitis / genetics
  • Esophagitis / pathology
  • Esophagoscopy
  • Esophagus / chemistry
  • Esophagus / drug effects*
  • Esophagus / pathology
  • Female
  • Fibrosis
  • Genotype
  • Humans
  • Immunohistochemistry
  • Infant
  • Lansoprazole
  • Male
  • Mucous Membrane / drug effects
  • Mucous Membrane / pathology
  • Phenotype
  • Placebo Effect
  • Polymorphism, Single Nucleotide
  • Promoter Regions, Genetic
  • Proton Pump Inhibitors / pharmacology
  • Transforming Growth Factor beta1 / analysis
  • Transforming Growth Factor beta1 / genetics
  • Treatment Outcome

Substances

  • 2-Pyridinylmethylsulfinylbenzimidazoles
  • Anti-Inflammatory Agents
  • Dosage Forms
  • Proton Pump Inhibitors
  • Transforming Growth Factor beta1
  • Lansoprazole
  • Budesonide

Associated data

  • ClinicalTrials.gov/NCT00638456