Oral Viscous Budesonide in Children With Eosinophilic Esophagitis After Repaired Esophageal Atresia: A Clinical Trial

被引：3

作者：

Tambucci, Renato ^{[1
,8
]}

Roversi, Marco ^{[2
]}

Rea, Francesca ^{[1
]}

Malamisura, Monica ^{[1
]}

Angelino, Giulia ^{[1
]}

Biondi, Isabella ^{[3
]}

Simeoli, Raffaele ^{[4
]}

Goffredo, Bianca Maria ^{[4
]}

Francalanci, Paola ^{[1
,5
]}

Simonetti, Alessandra ^{[3
]}

Livadiotti, Susanna ^{[3
]}

Corsetti, Tiziana ^{[6
]}

Dall'Oglio, Luigi ^{[7
]}

Rossi, Paolo ^{[3
]}

Pontrelli, Giuseppe ^{[3
]}

De Angelis, Paola ^{[1
]}

机构：

[1] Bambino Gesu Childrens Hosp IRCCS, Digest Endoscopy Unit, Rome, Italy

[2] Univ Roma Tor Vergata, Sch Pediat, Rome, Italy

[3] Bambino Gesu Childrens Hosp IRCCS, Ctr Excellence Dev & Implementat Med Vaccines Med, Rome, Italy

[4] Bambino Gesu Childrens Hosp IRCCS, Div Metab Dis & Drug Biol, Rome, Italy

[5] Bambino Gesu Childrens Hosp IRCCS, Dept Pathol, Rome, Italy

[6] Bambino Gesu Childrens Hosp IRCCS, Unit Clin Pharm, Rome, Italy

[7] Bambino Gesu Childrens Hosp IRCCS, Digest Endoscopy & Surg Unit, Rome, Italy

[8] Bambino Gesu Pediat Hosp, IRCCS, Digest Endoscopy Unit, Gastroenterol Hepatol & Nutr Dept, Piazza St Onofrio 4, I-00165 Rome, Italy

来源：

JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION | 2023年 / 77卷 / 02期

关键词：

Good Manufacturing Practice; phase 2 clinical trial; swallowed topical steroids; GUIDELINES; SUSPENSION; DIAGNOSIS; FEATURES;

D O I：

10.1097/MPG.0000000000003830

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

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 Gesu 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.

引用

页码：249 / 255

页数：7

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