Oral Viscous Budesonide as a First-Line Approach to Esophageal Stenosis in Epidermolysis Bullosa: an Open-Label Trial in Six Children

Andrea Zanini, Sophie Guez, Simona Salera, Giorgio Farris, Anna Morandi, Valerio Gentilino, Ernesto Leva, Francesca Manzoni, Maria Angela Pavesi, Susanna Esposito, Francesco Macchini

Research output: Contribution to journalArticle


Background: Esophageal and pharyngeal problems are common in the majority of patients with epidermolysis bullosa (EB). Repeated blister formation and ulceration, coupled with chronic inflammation, result in scarring and development of esophageal strictures.

Objective: This study aimed to evaluate whether oral viscous budesonide (OVB) was useful for treating esophageal structures in six pediatric patients (aged 8–17 years) with EB who were affected by dysphagia and esophageal strictures.

Methods: Patients were treated for 4 months with twice-daily oral budesonide nebulizer solution 0.5 mg/2 mL mixed with maltodextrin 5 g and artificial sweeteners.

Results: One patient developed a severe oral mycotic infection and discontinued treatment. The other five patients completed the treatment regimen and displayed significantly lower stricture indices (SIs) post-treatment (mean SI ± standard deviation 0.736 ± 0.101 pre-treatment versus 0.558 ± 0.162 post-treatment; p = 0.008). Patients experienced a mean SI decrease of 0.178 (range 0.026–0.296), as well as improved dietary habits in the absence of side effects.

Conclusion: These findings indicated that topical corticosteroids may significantly alleviate strictures in pediatric patients with EB, thereby limiting the need for endoscopic dilation and considerably improving patients’ quality of life.

Original languageEnglish
Pages (from-to)391-395
Number of pages5
JournalPediatric Drugs
Issue number5
Publication statusPublished - 2014


ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pharmacology (medical)
  • Medicine(all)

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