title: Primary prophylaxis of variceal hemorrhage in children with portal hypertension: a framework for future research.
abstract: Nonselective β-blocker therapy and endoscopic variceal ligation reduce the incidence of variceal hemorrhage in cirrhotic adults, but their use in children is controversial. There are no evidence-based recommendations for the prophylactic management of children at risk of variceal hemorrhage due to the lack of appropriate randomized controlled trials. In a recent gathering of experts at the American Association for the Study of Liver Diseases annual meeting, significant challenges were identified in attempting to design and implement a clinical trial of primary prophylaxis in children using either of these therapies. These challenges render such a trial unfeasible, primarily due to the large sample size required, inadequate knowledge of appropriate dosing of β-blockers, and difficulty in recruiting to a trial of endoscopic variceal ligation. Pediatric research should focus on addressing questions of natural history and diagnosis of varices, prediction of variceal bleeding, optimal approaches to β-blocker and ligation therapy, and alternative study designs to explore therapeutic efficacy in children.
authors.affiliation: Division of Gastroenterology, Hepatology & Nutrition, The Hospital for Sick Children, Toronto, Ontario, Canada. simon.ling@sickkids.ca
authors.forename: Simon C
authors.initials: SC
authors.lastname: Ling
authors.forename: Thomas
authors.initials: T
authors.lastname: Walters
authors.forename: Patrick J
authors.initials: PJ
authors.lastname: McKiernan
authors.forename: Kathleen B
authors.initials: KB
authors.lastname: Schwarz
authors.forename: Guadalupe
authors.initials: G
authors.lastname: Garcia-Tsao
authors.forename: Benjamin L
authors.initials: BL
authors.lastname: Shneider
mesh_terms: D000319:Adrenergic beta-Antagonists / Q000627:therapeutic use*; D000328:Adult; D002648:Child; D003131:Combined Modality Therapy; D004724:Endoscopy / Q000379:methods; D004932:Esophageal and Gastric Varices / Q000175:diagnosis / Q000188:drug therapy* / Q000601:surgery; D006471:Gastrointestinal Hemorrhage / Q000517:prevention & control*; D006801:Humans; D008026:Ligation
chemical_list: D000319:Adrenergic beta-Antagonists