Biliary Atresia in Children: Update on Disease Mechanism, Therapies, and Patient Outcomes

Overview

Journal Clin Liver Dis

Specialty Gastroenterology

Date 2022 Jul 22

PMID 35868678

Authors

Swati Antala

Sarah A Taylor

Affiliations

Soon will be listed here.

Abstract

Biliary atresia is a rare disease but remains the most common indication for pediatric liver transplantation as there are no effective medical therapies to slow progression after diagnosis. Variable contribution of genetic, immune, and environmental factors contributes to disease heterogeneity among patients with biliary atresia. Developing a deeper understanding of the disease mechanism will help to develop targeted medical therapies and improve patient outcomes.

Citing Articles

Predictors of outcome among children with biliary atresia: a single centre trial.

Ye C, Gao W PeerJ. 2025; 13:e19001.

PMID: 40017650 PMC: 11867042. DOI: 10.7717/peerj.19001.

Transcriptional analysis of murine biliary atresia identifies macrophage heterogeneity and subset-specific macrophage functions.

Gromer K, Chen S, Gadhvi G, Feng L, Shearn C, Antala S Front Immunol. 2025; 16:1506195.

PMID: 39949768 PMC: 11821939. DOI: 10.3389/fimmu.2025.1506195.

Interleukin 17A promotes glycolysis to activate human hepatic stellate cells by mediating the TRAF2/TRAF5/HuR/PFKFB3 axis.

Jiang T, Li S, Tang L, Tan Y, Ouyang W Cent Eur J Immunol. 2025; 49(4):404-424.

PMID: 39944257 PMC: 11811719. DOI: 10.5114/ceji.2024.145013.

Increased serum GM-CSF at diagnosis of biliary atresia is associated with improved biliary drainage.

Taylor S, Harpavat S, Gromer K, Andreev V, Loomes K, Bezerra J Pediatr Res. 2025; .

PMID: 39881181 DOI: 10.1038/s41390-025-03804-9.

Laparoscopic vs. open portoenterostomy for biliary atresia: a meta-analysis of pediatric surgical outcomes.

Zhu J, Wu B, Cai P, Pan J, Zhu Z Front Pediatr. 2024; 12:1476195.

PMID: 39717191 PMC: 11663641. DOI: 10.3389/fped.2024.1476195.

References

Shneider B, Brown M, Haber B, Whitington P, Schwarz K, Squires R . A multicenter study of the outcome of biliary atresia in the United States, 1997 to 2000. J Pediatr. 2006; 148(4):467-474. DOI: 10.1016/j.jpeds.2005.12.054. View

Tang K, Huang L, Huang Y, Lai C, Wu C, Wang S . Gamma-glutamyl transferase in the diagnosis of biliary atresia. Acta Paediatr Taiwan. 2008; 48(4):196-200. View

Zhao D, Zhou K, Chen Y, Xie W, Zhang Y . Development and validation of bile acid profile-based scoring system for identification of biliary atresia: a prospective study. BMC Pediatr. 2020; 20(1):255. PMC: 7251733. DOI: 10.1186/s12887-020-02169-8. View

Lages C, Simmons J, Maddox A, Jones K, Karns R, Sheridan R . The dendritic cell-T helper 17-macrophage axis controls cholangiocyte injury and disease progression in murine and human biliary atresia. Hepatology. 2016; 65(1):174-188. PMC: 5191928. DOI: 10.1002/hep.28851. View

Taylor S, Malladi P, Pan X, Wechsler J, Hulse K, Perlman H . Oligoclonal immunoglobulin repertoire in biliary remnants of biliary atresia. Sci Rep. 2019; 9(1):4508. PMC: 6418100. DOI: 10.1038/s41598-019-41148-7. View

Holterman A, Nguyen H, Nadler E, Vu G, Mohan P, Vu M . Granulocyte-colony stimulating factor GCSF mobilizes hematopoietic stem cells in Kasai patients with biliary atresia in a phase 1 study and improves short term outcome. J Pediatr Surg. 2021; 56(7):1179-1185. DOI: 10.1016/j.jpedsurg.2021.03.038. View

Decharun K, Leys C, West K, Finnell S . Prophylactic Antibiotics for Prevention of Cholangitis in Patients With Biliary Atresia Status Post-Kasai Portoenterostomy: A Systematic Review. Clin Pediatr (Phila). 2015; 55(1):66-72. DOI: 10.1177/0009922815594760. View

Berauer J, Mezina A, Okou D, Sabo A, Muzny D, Gibbs R . Identification of Polycystic Kidney Disease 1 Like 1 Gene Variants in Children With Biliary Atresia Splenic Malformation Syndrome. Hepatology. 2019; 70(3):899-910. PMC: 6642859. DOI: 10.1002/hep.30515. View

Gorgis N, Kennedy C, Lam F, Thompson K, Coss-Bu J, Arikan A . Clinical Consequences of Cardiomyopathy in Children With Biliary Atresia Requiring Liver Transplantation. Hepatology. 2018; 69(3):1206-1218. DOI: 10.1002/hep.30204. View

10.

Muraji T, Harada T, Miki K, Moriuchi T, Obatake M, Tsugawa C . Urinary sulfated bile acid concentrations in infants with biliary atresia and breast-feeding jaundice. Pediatr Int. 2003; 45(3):281-3. DOI: 10.1046/j.1442-200x.2003.01710.x. View

11.

Mack C, Anderson K, Aubrey M, Rosenthal P, Sokol R, Freed B . Lack of HLA predominance and HLA shared epitopes in biliary Atresia. Springerplus. 2013; 2(1):42. PMC: 3595468. DOI: 10.1186/2193-1801-2-42. View

12.

Okazaki T, Ochi T, Nakamura H, Tsukui T, Koga H, Urao M . Needle liver biopsy has potential for delaying Kasai portoenterostomy and Is obsolete for diagnosing biliary atresia in the laparoscopic era. J Pediatr Surg. 2019; 54(12):2570-2573. DOI: 10.1016/j.jpedsurg.2019.08.028. View

13.

Sullivan J, Sundaram S, Pan Z, Sokol R . Parenteral nutrition supplementation in biliary atresia patients listed for liver transplantation. Liver Transpl. 2011; 18(1):120-8. PMC: 3245380. DOI: 10.1002/lt.22444. View

14.

Schreiber R, Barker C, Roberts E, Martin S, Alvarez F, Smith L . Biliary atresia: the Canadian experience. J Pediatr. 2007; 151(6):659-65, 665.e1. DOI: 10.1016/j.jpeds.2007.05.051. View

15.

Kobayashi H, Puri P, OBriain D, Surana R, Miyano T . Hepatic overexpression of MHC class II antigens and macrophage-associated antigens (CD68) in patients with biliary atresia of poor prognosis. J Pediatr Surg. 1997; 32(4):590-3. DOI: 10.1016/s0022-3468(97)90714-4. View

16.

Schwab I, Nimmerjahn F . Intravenous immunoglobulin therapy: how does IgG modulate the immune system?. Nat Rev Immunol. 2013; 13(3):176-89. DOI: 10.1038/nri3401. View

17.

Lam W, Tang C, So M, Yue H, Hsu J, Chung P . Identification of a wide spectrum of ciliary gene mutations in nonsyndromic biliary atresia patients implicates ciliary dysfunction as a novel disease mechanism. EBioMedicine. 2021; 71:103530. PMC: 8403738. DOI: 10.1016/j.ebiom.2021.103530. View

18.

Hopkins P, Yazigi N, Nylund C . Incidence of Biliary Atresia and Timing of Hepatoportoenterostomy in the United States. J Pediatr. 2017; 187:253-257. DOI: 10.1016/j.jpeds.2017.05.006. View

19.

Lopez R, Ooi C, Krishnan U . Early and Peri-operative Prognostic Indicators in Infants Undergoing Hepatic Portoenterostomy for Biliary Atresia: a Review. Curr Gastroenterol Rep. 2017; 19(4):16. DOI: 10.1007/s11894-017-0555-z. View

20.

Urushihara N, Iwagaki H, Yagi T, Kohka H, KOBASHI K, Morimoto Y . Elevation of serum interleukin-18 levels and activation of Kupffer cells in biliary atresia. J Pediatr Surg. 2000; 35(3):446-9. DOI: 10.1016/s0022-3468(00)90211-2. View