Oral Viscous Budesonide in Children With Eosinophilic Esophagitis After Repaired Esophageal Atresia
A Clinical Trial
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 authorMarco Roversi MD
Residency School of Pediatrics, University of Rome Tor Vergata, Rome, Italy
Search for more papers by this authorFrancesca Rea MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorMonica Malamisura MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorGiulia Angelino MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorIsabella 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
Search for more papers by this authorRaffaele Simeoli PharmD, PhD
Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorBianca Maria Goffredo MSc
Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorPaola Francalanci MD
Department of Pathology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorAlessandra 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
Search for more papers by this authorSusanna 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
Search for more papers by this authorTiziana Corsetti PharmD
Unit of Clinical Pharmacy, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorLuigi Dall’Oglio MD
Digestive Endoscopy and Surgery Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorPaolo 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
Search for more papers by this authorGiuseppe 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
Search for more papers by this authorPaola De Angelis MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorCorresponding 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 authorMarco Roversi MD
Residency School of Pediatrics, University of Rome Tor Vergata, Rome, Italy
Search for more papers by this authorFrancesca Rea MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorMonica Malamisura MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorGiulia Angelino MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorIsabella 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
Search for more papers by this authorRaffaele Simeoli PharmD, PhD
Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorBianca Maria Goffredo MSc
Division of Metabolic Diseases and Drug Biology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorPaola Francalanci MD
Department of Pathology, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorAlessandra 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
Search for more papers by this authorSusanna 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
Search for more papers by this authorTiziana Corsetti PharmD
Unit of Clinical Pharmacy, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorLuigi Dall’Oglio MD
Digestive Endoscopy and Surgery Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorPaolo 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
Search for more papers by this authorGiuseppe 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
Search for more papers by this authorPaola De Angelis MD
Digestive Endoscopy Unit, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Search for more papers by this authorThe 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.
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