A phase 2 trial of short-term intravenous N-acetylcysteine in biliary atresia after Kasai portoenterostomy.

ABSTRACT:

Background

For infants with biliary atresia, the only treatment that can establish bile flow and delay need for liver transplant is the Kasai portoenterostomy (KP). Unfortunately, the KP has variable success. In this study, we hypothesized that intravenous N-acetylcysteine (IV NAC) treatment following KP would improve bile flow.

Methods

This was a phase 2 study following the two-stage "minimax" trial design. Participants received IV NAC (150 mg/kg/day) for 7 days after KP, and the primary endpoint was achieving total serum bile acids (TSBA) ≤10 μmol/L within 24 weeks of KP. Secondary endpoints were clinical markers and the occurrence of sentinel events.

Results

There were 12 participants in stage 1 who received treatment, with none achieving TSBAs ≤10 μmol/L within 24

SUBMITTER: Harpavat S 

PROVIDER: S-EPMC12150978 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature