https://www.ncbi.nlm.nih.gov/pubmed/30664564 Biliary atresia

J Pediatr Gastroenterol Nutr. 2019 Jan 17. doi: 10.1097/MPG.0000000000002256. [Epub ahead of print]
A Phase I/IIa Trial of Intravenous Immunoglobulin Following Portoenterostomy in Biliary Atresia.

Mack CL1, Spino C2, Alonso EM3, Bezerra JA4, Moore J2, Goodhue C5, Ng VL6, Karpen SJ7, Venkat V8, Loomes KM9, Wang K5, Sherker AH10, Magee JC2, Sokol RJ1; and The ChiLDReN Network.

Abstract

OBJECTIVES:
Biliary atresia (BA) is a progressive neonatal fibroinflammatory cholangiopathy. We hypothesized that intravenous immunoglobulin (IVIg) would be safe, feasible, acceptable and efficacious for the treatment of BA. The primary objective of this study was to establish the feasibility, acceptability and safety profile of IVIg administration after hepatoportoenterostomy (HPE) in BA. The secondary objective was to determine the treatment efficacy of IVIg based on good bile drainage and survival with the native liver.

METHODS:
A multi-center, prospective, open-labeled, phase I/IIA trial of IVIg was conducted, with 1 gm/kg/dose of IVIg infused at 3-5 days, 30 days and 60 days post-HPE, and subjects followed for 360 days post-HPE. Twenty-nine participants completed the study.

RESULTS:
Administration of IVIg infusions was feasible and acceptable in 79%. None of the serious adverse events (SAEs) were directly related to IVIg infusions, however 90% of participants had an SAE. Compared to a historical placebo-arm group, there was no significant increase in the proportion of IVIg participants with a serum total bilirubin < 1.5 mg/dL at 90, 180, or 360 days post-HPE. Survival with the native liver in the IVIg participants showed no significant benefit over the historical placebo-arm, with a difference at 360 days of -11.9% (IVIg:58.6%, placebo:70.5%; 90% UCB:2.1%; p > 0.05).

CONCLUSIONS:
Although IVIg infusions in infants with BA post-HPE were feasible, acceptable and safe, there was no trend to lower bilirubin levels or improved 360 day survival with the native liver.