title: Oral viscous budesonide as a first-line approach to esophageal stenosis in epidermolysis bullosa: an open-label trial in six children.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Pediatric Surgery Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Zanini
authors.forename: Sophie
authors.initials: S
authors.lastname: Guez
authors.forename: Simona
authors.initials: S
authors.lastname: Salera
authors.forename: Giorgio
authors.initials: G
authors.lastname: Farris
authors.forename: Anna
authors.initials: A
authors.lastname: Morandi
authors.forename: Valerio
authors.initials: V
authors.lastname: Gentilino
authors.forename: Ernesto
authors.initials: E
authors.lastname: Leva
authors.forename: Francesca
authors.initials: F
authors.lastname: Manzoni
authors.forename: Maria Angela
authors.initials: MA
authors.lastname: Pavesi
authors.forename: Susanna
authors.initials: S
authors.lastname: Esposito
authors.forename: Francesco
authors.initials: F
authors.lastname: Macchini
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage* / Q000009:adverse effects; D019819:Budesonide / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D004820:Epidermolysis Bullosa / Q000188:drug therapy*; D004940:Esophageal Stenosis / Q000188:drug therapy*; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage* / Q000009:adverse effects; D006801:Humans; D008297:Male; D011788:Quality of Life
chemical_list: D000893:Anti-Inflammatory Agents; D005938:Glucocorticoids; D019819:Budesonide