Primary Prophylaxis of Variceal Hemorrhage in Children With Portal Hypertension: A Framework for Future Research

Nonselective beta-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 beta-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 beta-blocker and ligation therapy, and alternative study designs to explore therapeutic efficacy in children.