A phase 2 trial of N-Acetylcysteine in Biliary atresia after Kasai portoenterostomy.

ABSTRACT:

Background

Biliary atresia (BA) is a life-threatening liver disease of infancy, characterized by extrahepatic biliary obstruction, bile retention, and progressive liver injury. The Kasai portoenterostomy (KP) is BA's only nontransplant treatment. Its success is variable and depends on restoration of hepatic bile flow. Many adjunctive therapeutics have been studied to improve outcomes after the KP, but none demonstrate effectiveness. This study tests if N-acetylcysteine (NAC), a precursor to the choleretic glutathione, improves bile flow after KP.

Methods

This report describes the design of an open-label, single center, Phase 2 study to determine the effect of NAC following KP on markers of bile flow and outcomes in BA. The intervention is intravenous NAC (150 mg/kg/day) admi

SUBMITTER: Tessier MEM 

PROVIDER: S-EPMC6542754 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature