Emerging treatments
Corticosteroids post hepatoportoenterostomy
Corticosteroids have anti-inflammatory and immunomodulatory characteristics that are thought to increase bile salt-independent bile flow. Meta-analysis of two randomised controlled trials failed to provide sufficient evidence to determine the effect of corticosteroids, compared with placebo, following hepatoportoenterostomy in infants with biliary atresia.[86] Multicentre randomised controlled trials of high methodological quality are required.
Ileal bile acid transporter (IBAT) inhibitors
IBAT inhibitors are minimally absorbed oral agents that medically interrupt the enterohepatic circulation by blocking bile acid reuptake in the distal ileum.[87] Two drugs in this class, maralixibat and odevixibat, have been approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for treatment of cholestatic pruritus in inherited paediatric cholestatic liver diseases (Alagille syndrome and progressive familial intrahepatic cholestasis). These drugs have been studied in randomised, placebo controlled clinical trials in infants with biliary atresia, based on the hypothesis that increased elimination of bile acids will improve clinical outcomes post-hepatoportoenterostomy in biliary atresia.[88][89] Results are not yet available from the odevixibat trial. The maralixibat trial was terminated as it showed no benefit for the outcomes assessed, including its primary outcome of change in serum bilirubin levels at 6 months post-hepatoportoenterostomy.[88]
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