Volume 77, Issue 2 pp. 249-255
Original Article: Gastroenterology: Eosinophilic Gastrointestinal Disorders

Oral Viscous Budesonide in Children With Eosinophilic Esophagitis After Repaired Esophageal Atresia

A Clinical Trial

Renato Tambucci MD, PhD

Corresponding Author

Renato Tambucci MD, PhD

Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

Address correspondence and reprint requests to Renato Tambucci, MD, PhD, Digestive Endoscopy Unit, Gastroenterology, Hepatology and Nutrition Department, Bambino Gesù Children’s Hospital, IRCCS, Piazza Sant’Onofrio 4, Rome 00165, Italy (e-mail: [email protected], [email protected]).Search for more papers by this author
Marco Roversi MD

Marco Roversi MD

Residency School of Pediatrics, University of Rome Tor Vergata, Rome, Italy

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Francesca Rea MD

Francesca Rea MD

Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Monica Malamisura MD

Monica Malamisura MD

Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Giulia Angelino MD

Giulia Angelino MD

Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Isabella Biondi MSc

Isabella Biondi MSc

Centre of Excellence for the Development and Implementation of Medicines, Vaccines, and Medical Devices for Pediatric Use, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Raffaele Simeoli PharmD, PhD

Raffaele Simeoli PharmD, PhD

Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Bianca Maria Goffredo MSc

Bianca Maria Goffredo MSc

Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Paola Francalanci MD

Paola Francalanci MD

Department of Pathology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Alessandra Simonetti MD

Alessandra Simonetti MD

Centre of Excellence for the Development and Implementation of Medicines, Vaccines, and Medical Devices for Pediatric Use, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Susanna Livadiotti MD

Susanna Livadiotti MD

Centre of Excellence for the Development and Implementation of Medicines, Vaccines, and Medical Devices for Pediatric Use, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Tiziana Corsetti PharmD

Tiziana Corsetti PharmD

Unit of Clinical Pharmacy, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Luigi Dall’Oglio MD

Luigi Dall’Oglio MD

Digestive Endoscopy and Surgery Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Paolo Rossi MD, PhD

Paolo Rossi MD, PhD

Centre of Excellence for the Development and Implementation of Medicines, Vaccines, and Medical Devices for Pediatric Use, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Giuseppe Pontrelli MD, PhD

Giuseppe Pontrelli MD, PhD

Centre of Excellence for the Development and Implementation of Medicines, Vaccines, and Medical Devices for Pediatric Use, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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Paola De Angelis MD

Paola De Angelis MD

Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy

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First published: 17 May 2023
Citations: 3

The authors report no conflicts of interest.

Supplemental digital content is available for this article. Direct URL citations appear in the printed text, and links to the digital files are provided in the HTML text of this article on the journal’s Web site (www.jpgn.org).

URL (website address) and trial identification number: https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-002691-14/IT; EudraCT number: 2019-002691-14.

Sources of Funding: This work was supported by the Italian Ministry of Health.

R.T. and M.R. contributed equally to the article.

Abstract

Objectives:

A high prevalence of eosinophilic esophagitis (EoE) has been reported in children with repaired esophageal atresia (EA). Topical steroids proved to be an effective and safe therapy in EoE, although not approved in pediatrics. We report the results of the first clinical trial of oral viscous budesonide (OVB) performed in children with EoE after repaired esophageal atresia (EoE-EA).

Methods:

This open-label, single-arm, phase 2 clinical trial with randomized pharmacokinetic sampling, was conducted at the Bambino Gesù Children’s Hospital between September 2019 and June 2021. EoE-EA patients received an age-banded dose of OVB twice daily for 12 weeks and were endoscopically evaluated. The primary endpoint was the rate of patients achieving histological remission. Secondary endpoints included clinical and endoscopic benefit after treatment, and safety assessments.

Results:

Eight consecutive EA-EoE patients were enrolled (median age 9.1 years, interquartile range 5.5). Of these, 5 received 0.8 mg and 3 received 1.0 mg twice daily of OVB. Histological remission was obtained in all but 1 patient (87.5%). The clinical score showed significant improvement at the end of treatment in all patients. No endoscopic features of EoE were found after treatment. No treatment-emergent adverse event occurred.

Conclusion:

OVB is an effective, safe, and well-tolerated formulation of budesonide for use in pediatric patients with EoE-EA.

The full text of this article hosted at iucr.org is unavailable due to technical difficulties.