Literature DB >> 25138121

Oral viscous budesonide as a first-line approach to esophageal stenosis in epidermolysis bullosa: an open-label trial in six children.

Andrea Zanini1, Sophie Guez, Simona Salera, Giorgio Farris, Anna Morandi, Valerio Gentilino, Ernesto Leva, Francesca Manzoni, Maria Angela Pavesi, Susanna Esposito, Francesco Macchini.   

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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Year:  2014        PMID: 25138121     DOI: 10.1007/s40272-014-0086-0

Source DB:  PubMed          Journal:  Paediatr Drugs        ISSN: 1174-5878            Impact factor:   3.022


  15 in total

1.  Colon interposition for esophageal stenosis in a patient with epidermolysis bullosa.

Authors:  B Demiroğullari; K Sönmez; Z Türkyilmaz; B Altuntaş; R Karabulut; A C Başaklar; N Kale
Journal:  J Pediatr Surg       Date:  2001-12       Impact factor: 2.545

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

Authors:  Ranjan Dohil; Robert Newbury; Lyman Fox; John Bastian; Seema Aceves
Journal:  Gastroenterology       Date:  2010-05-07       Impact factor: 22.682

Review 3.  Extracutaneous manifestations and complications of inherited epidermolysis bullosa: part I. Epithelial associated tissues.

Authors:  Jo-David Fine; Jemima E Mellerio
Journal:  J Am Acad Dermatol       Date:  2009-09       Impact factor: 11.527

4.  Oral viscous budesonide therapy in children with epidermolysis bullosa and proximal esophageal strictures.

Authors:  R Dohil; S S Aceves; M A Dohil
Journal:  J Pediatr Gastroenterol Nutr       Date:  2011-06       Impact factor: 2.839

5.  Anastomotic strictures and endoscopic dilatations following esophageal atresia repair.

Authors:  Filippo Parolini; Ernesto Leva; Anna Morandi; Francesco Macchini; Valerio Gentilino; Antonio Di Cesare; Maurizio Torricelli
Journal:  Pediatr Surg Int       Date:  2013-03-22       Impact factor: 1.827

6.  Management of esophageal spasm in epidermolysis bullosa dystrophica using verapamil.

Authors:  J D Mitchell; M Eisenberg
Journal:  J Pediatr Gastroenterol Nutr       Date:  1989-01       Impact factor: 2.839

Review 7.  Inhaled corticosteroids in children: effects on bone mineral density and growth.

Authors:  Anne L Fuhlbrigge; H William Kelly
Journal:  Lancet Respir Med       Date:  2014-04-08       Impact factor: 30.700

8.  Management of esophageal stenosis in recessive dystrophic epidermolysis bullosa.

Authors:  G E Feurle; H Weidauer; G Baldauf; T Schulte-Braucks; I Anton-Lamprecht
Journal:  Gastroenterology       Date:  1984-12       Impact factor: 22.682

9.  Recessive dystrophic epidermolysis bullosa treated with phenytoin.

Authors:  A A Abahussein; A A al-Zayir; W Z Mostafa; A N Okoro
Journal:  Int J Dermatol       Date:  1992-10       Impact factor: 2.736

10.  Chronic recurrent esophageal strictures treated with balloon dilation in children with autosomal recessive epidermolysis bullosa dystrophica.

Authors:  M B Heyman; M Zwass; M Applebaum; C D Rudolph; R Gordon; E J Ring
Journal:  Am J Gastroenterol       Date:  1993-06       Impact factor: 10.864

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  6 in total

Review 1.  [Mucosal manifestations of epidermolysis bullosa : Clinical presentation and management].

Authors:  C Prodinger; A Diem; J W Bauer; M Laimer
Journal:  Hautarzt       Date:  2016-10       Impact factor: 0.751

Review 2.  [Hereditary epidermolysis bullosa in school children and adolescents. Clinical picture and interdisciplinary management].

Authors:  H Ott; C Eich; K Schriek; B Ludwikowski
Journal:  Hautarzt       Date:  2016-04       Impact factor: 0.751

3.  Approach and Safety of Esophageal Dilation for Treatment of Strictures in Children With Epidermolysis Bullosa.

Authors:  Bradley T Anderson; James A Feinstein; Robert E Kramer; Michael R Narkewicz; Anna L Bruckner; David E Brumbaugh
Journal:  J Pediatr Gastroenterol Nutr       Date:  2018-12       Impact factor: 2.839

Review 4.  Endoscopic management of esophageal stenosis in children: New and traditional treatments.

Authors:  Luigi Dall'Oglio; Tamara Caldaro; Francesca Foschia; Simona Faraci; Giovanni Federici di Abriola; Francesca Rea; Erminia Romeo; Filippo Torroni; Giulia Angelino; Paola De Angelis
Journal:  World J Gastrointest Endosc       Date:  2016-02-25

Review 5.  Emergency management in epidermolysis bullosa: consensus clinical recommendations from the European reference network for rare skin diseases.

Authors:  Jemima E Mellerio; Maya El Hachem; Nathalia Bellon; Giovanna Zambruno; Hana Buckova; Rudolf Autrata; Carmen Salavastru; Tamara Caldaro; Celine Greco; Cristina Has; Christine Bodemer
Journal:  Orphanet J Rare Dis       Date:  2020-06-06       Impact factor: 4.123

Review 6.  Small molecule drug development for rare genodermatoses - evaluation of the current status in epidermolysis bullosa.

Authors:  Verena Wally; Manuela Reisenberger; Sophie Kitzmüller; Martin Laimer
Journal:  Orphanet J Rare Dis       Date:  2020-10-19       Impact factor: 4.123

  6 in total

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