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

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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.

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.

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Acknowledgments

This study was supported by a grant from the Italian Ministry of Health (Bando Ricerca Corrente 2014 850/01). The authors declare that they have no conflicts of interest that are directly relevant to the content of this study.

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Authors and Affiliations

  1. Pediatric Surgery Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

    Andrea Zanini, Giorgio Farris, Anna Morandi, Valerio Gentilino, Ernesto Leva & Francesco Macchini

  2. Pediatric Highly Intensive Care Unit, Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Via Commenda 9, 20122, Milan, Italy

    Sophie Guez, Simona Salera, Francesca Manzoni & Susanna Esposito

  3. Pediatric Radiology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

    Maria Angela Pavesi

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  1. Andrea Zanini
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  2. Sophie Guez
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  3. Simona Salera
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  4. Giorgio Farris
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  5. Anna Morandi
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  6. Valerio Gentilino
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  7. Ernesto Leva
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  8. Francesca Manzoni
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  9. Maria Angela Pavesi
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  10. Susanna Esposito
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  11. Francesco Macchini
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Corresponding author

Correspondence to Susanna Esposito.

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Zanini, A., Guez, S., Salera, S. et al. Oral Viscous Budesonide as a First-Line Approach to Esophageal Stenosis in Epidermolysis Bullosa: an Open-Label Trial in Six Children. Pediatr Drugs 16, 391–395 (2014). https://doi.org/10.1007/s40272-014-0086-0

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  • DOI: https://doi.org/10.1007/s40272-014-0086-0

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