Explore chapters and articles related to this topic
Liver transplantation
Published in Mark Davenport, James D. Geiger, Nigel J. Hall, Steven S. Rothenberg, Operative Pediatric Surgery, 2020
Caroline Lemoine, Riccardo A. Superina
Chronic liver disease with cirrhosis is the most common indication for LT in children, and biliary atresia is the most common form of chronic liver disease leading to transplantation, accounting for approximately one-third of liver transplants in most series. Most children with biliary atresia have already undergone a Kasai portoenterostomy in the first 2–10 weeks of life, with failure characterized by progressive jaundice, increasing portal hypertension, ascites, and malnutrition. Even those with initial clearance of jaundice may still require LT later. Chronic liver disease may also lead to progressive encephalopathy, metabolic bone disease, intractable pruritus, pulmonary hypertension, and hepatopulmonary syndrome. There are no uniformly accurate predictors of success following the Kasai procedure, but some infants will derive no benefit in terms of relief of jaundice and will require LT within 1–2 years of life. Those who are diagnosed late with biliary atresia may benefit from a primary transplant.
Alagille Syndrome
Published in Dongyou Liu, Handbook of Tumor Syndromes, 2020
For patients with cholestasis, supportive treatment with choleretic agents (e.g., ursodeoxycholic acid, naltrexone, rifampin, colesevelam, and cholestyramine) helps ameliorate debilitating pruritus and disfiguring xanthomas. Surgical partial internal biliary diversion and ileal exclusion are also helpful in controlling itch and xanthomas. Kasai procedure (hepatic portoenterostomy), which is often used in patients with biliary atresia, seems to worsen the outcome of ALGS. Liver transplantation may be considered for end-stage liver disease (about 15% of ALGS patients, who tend to have a high serum total bilirubin between 12–24 months of age together with fibrosis on liver biopsy and xanthomata on physical examination), as it improves liver function and catch-up growth in 90% of cases and has a 5-year survival rate of 80% [26].
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
Outcome for biliary atresia patients treated at a low-volume centre
Published in Scandinavian Journal of Gastroenterology, 2018
Kristin Bjørnland, Maren Hinna, Gunnar Aksnes, Kjetil Juul Stensrud, Kjetil Ertresvåg, Anniken Bjørnstad-Østensen, Truls Sanengen, Pål-Dag Line, Ingegerd Aagenæs, Lars Aabakken, Ragnhild Emblem, Runar Almaas
Forty-five babies, 29 girls (64%), were diagnosed with BA during the study period. Weight at admittance was median 4600 (1935–5980) grams, and total and conjugated plasma bilirubin were median 162 (72–352) and 110 (25–216) μmol/l, respectively. The median age at last follow-up was 86 (4–194) months. Three patients died at the age of 18, 26 and 51 months, respectively; all had been operated with a Kasai procedure. One of the patients died of causes unrelated to BA with normal liver function at the age of 26 months, whereas the two other patients died 15 and 46 months after liver transplantation.
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.
Hepatocyte growth factor levels in livers and serum at Kasai-portoenterostomy are not predictive of clinical outcome in infants with biliary atresia
Published in Growth Factors, 2019
Omid Madadi-Sanjani, Joachim F. Kuebler, Stephanie Dippel, Anna Gigina, Christine S. Falk, Gertrud Vieten, Claus Petersen, Christian Klemann
For future studies we therefore need a synergism of basic research and clinical trials, to get more information on the pathophysiology of the deterioration processes in BA and in the meantime improve the outcomes of Kasai procedure with evidence-based pre-, peri-, and postoperative methods, e.g. the proven benefit of centralization for rare diseases and the recently discussed postoperative steroid and antibiotic use (Madadi-Sanjani and Petersen 2018).