Treatment of paediatric cholestasis due to canalicular transport defects: yet another step forward

Paediatric cholestasis is a rare but devastating group of diseases that usually manifest in infancy or childhood by impaired bile secretion, due to either primary or secondary alterations affecting the liver secretory machinery, and that may progress to cirrhosis and liver failure. Although there is resemblance in clinical symptoms, the cause of these diseases can be completely different, which is important because it determines, among others, the response to treatment. For a specific subgroup of cholestatic diseases, namely those associated with canalicular transport defects, our pathophysiological understanding has been increased enormously over the last decade. In parallel, treatment options have been developed,1 and the article by Gordo–Gilart et al2 yet takes another step forward. Several transport proteins located at the canalicular membrane of hepatocytes are required to maintain the correct secretory function of the liver (figure 1). A crucial role is played by...

Source: Gut - December 8, 2014 Category: Gastroenterology Authors: Marin, J. J. G., Houwen, R. H. J. Tags: Commentary Source Type: research