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Biliary Atresia
Published in Gianfranco Alpini, Domenico Alvaro, Marco Marzioni, Gene LeSage, Nicholas LaRusso, The Pathophysiology of Biliary Epithelia, 2020
Several approaches have been used to attempt to enhance adequate bile drainage after the Kasai procedure including the use of choleretics, such as ursodeoxycholic acid and phenobarbital, bile aid binding resins, and anti-inflammatory drugs, even corticosteroids.47,48 In Japan, patients are treated post-operatively with a protocol that includes intravenous dehydrocholic acid, oral ursodeoxycholic acid and intravenous methylprednisolone. The patients are then treated with oral prednisone every other day for 2 months.49 Unfortunately, due to the limited nature with which these approaches have been studied, there is no evidence that they are successful. Many approaches have also been used to reduce the likelihood of cholangitis/sepsis after the hepatoportoenterostomy. Patients have been treated with postoperative intravenous antibiotics and others with long-term prophylactic oral antibiotics. The Roux-en-Y limb of the portoenterostomy has been lengthened, exteriorized or made into a valved conduit. None of these interventions has any substantive proven benefit. Ultimately only 10–20% of the patients who undergo the Kasai procedure will have sustained relief of biliary obstruction.43–45
Odevixibat: an investigational inhibitor of the ileal bile acid transporter (IBAT) for the treatment of biliary atresia
Published in Expert Opinion on Investigational Drugs, 2022
Biliary atresia (BA) is a condition presenting with the onset of severe cholestasis in the first months of life. Although immunological, infectious and genetic factors have been investigated, the cause of ascending bile duct sclerosis in infants remains unclear [1]. Incidence varies worldwide, ranging from about 1 in 18,000 live births in Europe to a higher incidence in Asian countries, e.g. in Taiwan with 1 in 6,200 [2–4]. Typical symptoms are prolonged jaundice (conjugated hyperbilirubinemia), discolored (acholic) stools, and hepatomegaly. As the disease progresses, typical symptoms are failure to thrive, pruritus, and signs of portal hypertension with splenomegaly, ascites and esophageal variceal bleeding. Without treatment, this can lead to death within the first two years of life [5]. Kasai hepatoportoenterostomy, first performed in 1959, aims to restore bile flow [6]. During the operation, a Roux-en-Y anastomosis of the small intestine is placed in the area of the porta hepatis, following resection of the fibrotic area of the liver capsule, where the intrahepatic biliary tract normally connects to the common hepatic duct. The success of the Kasai procedure, i.e. the restoration and maintenance of bile flow from the liver, impacts on the indication for liver transplantation as well as complications, such as cholangitis or esophageal variceal bleeding [1]. Even when the Kasai procedure is optimally performed within the first 45 days of life, affected children often have ongoing liver damage, leading to cirrhosis due to accumulation of bile acids. Around 50% of children require liver transplantation by their 2nd birthday, only 20% reach adulthood with their own liver [7]. Therefore, BA is the leading cause of liver transplantation in childhood [8], especially in younger patients [9]. The primary goals of medical treatment at this early age are to support the child’s growth and to monitor the onset of complications.