Medical Status of 219 Children with Biliary Atresia Surviving Long-Term with Their Native Livers: Results from a North American Multicenter Consortium

Ng, Vicky L.; Haber, Barbara; Magee, John C.; Miethke, Alexander; Murray, Karen F.; Michail, Sonia; Karpen, Saul J.; Kerkar, Nanda; Molleston, Jean P.; Romero, René; Rosenthal, Philip; Schwarz, Kathleen B.; Shneider, Benjamin L.; Turmelle, Yumirle P.; Alonso, Estella M.; Sherker, Averell H.; Sokol, Ronald J.

doi:10.1016/j.jpeds.2014.05.038

Cited by 75 publications

(91 citation statements)

References 47 publications

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“…Recent reports indicate that those who develop bile flow (defined as a serum total bilirubin level <2 mg/dL) within 3 months of HPE have a >80% chance to be in the group of BA patients surviving with the native liver at 2 years of age . More importantly, although these patients are considered “successful” responders to Kasai HPE, at least 75% will develop ongoing fibrosis, portal hypertension, and cirrhosis, with significant risks of hepatic complications, death, and transplant from childhood through young adulthood . These BA patients are candidates for trials of an array of therapeutic agents as they have the potential to respond and stand to benefit significantly.…”

Section: Innovative Approaches To Treatmentmentioning

confidence: 99%

Biliary Atresia: Clinical and Research Challenges for the Twenty‐First Century

et al. 2018

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Biliary atresia (BA) is a fibroinflammatory disease of the intra- and extrahepatic biliary tree. Without medical treatment, surgical hepatic portoenterosmy (HPE) may restore bile drainage, but progression of the intrahepatic disease results in complications of portal hypertension and advanced cirrhosis in most children. Recognizing that further progress in the field is unlikely without a better understanding of the underlying cause(s) and pathogenesis of the disease, the National Institutes of Diabetes and Digestive and Kidney Diseases sponsored a research workshop focused on innovative and promising approaches and on identifying future areas of research. Investigators discussed recent advances using gestational ultrasound and results of newborn BA screening with serum direct (conjugated) bilirubin that support a pre-natal onset of biliary injury. Experimental and human studies implicate the toxic properties of environmental toxins (e.g. biliatresone) and of viruses (e.g. CMV) to the biliary system. Among host factors, sequence variants in genes related to biliary development and ciliopathies, a notable lack of a cholangiocyte glycocalyx and of submucosal collagen bundles in the neonatal extrahepatic bile ducts, and an innate pro-inflammatory bias of the neonatal immune system contribute to an increased susceptibility to damage and obstruction following an epithelial injury. These advances form the foundation for a future research agenda focused on identifying the environmental and host factor(s) that cause BA, the potential use of population screening, studies of the mechanisms of prominent fibrosis in young infants, determinations of clinical surrogates of disease progression, and the design of clinical trials that target subgroups of patients with initial drainage following HPE. This article is protected by copyright. All rights reserved.

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Section: Innovative Approaches To Treatmentmentioning

confidence: 99%

Biliary Atresia: Clinical and Research Challenges for the Twenty‐First Century

et al. 2018

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“…Most patients in their cohort experienced multiple episodes of cholangitis requiring hospitalization, with an average length of stay of 15 days. Ng et al 25 reported that 17% of 219 patients who retained their native livers at least 5 years after their Kasai operations had experienced an episode of cholangitis in the preceding year.…”

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confidence: 99%

Newborn Screening for Biliary Atresia

Moss¹,

Caty²,

Davidoff³

et al. 2015

Pediatrics

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Biliary atresia is the most common cause of pediatric end-stage liver disease and the leading indication for pediatric liver transplantation. Affected infants exhibit evidence of biliary obstruction within the first few weeks after birth. Early diagnosis and successful surgical drainage of bile are associated with greater survival with the child’s native liver. Unfortunately, because noncholestatic jaundice is extremely common in early infancy, it is difficult to identify the rare infant with cholestatic jaundice who has biliary atresia. Hence, the need for timely diagnosis of this disease warrants a discussion of the feasibility of screening for biliary atresia to improve outcomes. Herein, newborn screening for biliary atresia in the United States is assessed by using criteria established by the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children. Published analyses indicate that newborn screening for biliary atresia by using serum bilirubin concentrations or stool color cards is potentially life-saving and cost-effective. Further studies are necessary to evaluate the feasibility, effectiveness, and costs of potential screening strategies for early identification of biliary atresia in the United States.

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“…Cholangitis is commonly seen early after the Kasai surgery. The majority of the patients surviving into adulthood without a transplant do not develop cholangitis and are not on any antibiotic prophylaxis [56]. Cholangitis, if it occurs, should be treated appropriately with intravenous or oral antibiotics depending on the clinical and biochemical severity at presentation.…”

Section: Biliary Atresiamentioning

confidence: 99%

The management of childhood liver diseases in adulthood

Joshi

Gupta

Samyn

et al. 2017

Journal of Hepatology

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SummaryAn increasing number of patients with childhood liver disease survive into adulthood. These young adults are now entering adult services and require ongoing management. Aetiologies can be divided into liver diseases that develop in young adults which present to adult hepatologists i.e., biliary atresia and Alagille syndrome or liver diseases that occur in children/adolescents and adults i.e., autoimmune hepatitis or Wilson's disease. To successfully manage these young adults, a dynamic and responsive transition service is essential. In this review, we aim to describe the successful components of a transition service highlighting the importance of self-management support and a multi-disciplinary approach. We will also review some of the liver specific aetiologies which are unique to young adults, offering an update on pathogenesis, management and outcomes. Ó

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Medical Status of 219 Children with Biliary Atresia Surviving Long-Term with Their Native Livers: Results from a North American Multicenter Consortium

Cited by 75 publications

References 47 publications

Biliary Atresia: Clinical and Research Challenges for the Twenty‐First Century

Biliary Atresia: Clinical and Research Challenges for the Twenty‐First Century

Newborn Screening for Biliary Atresia

The management of childhood liver diseases in adulthood

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