Recent Research Summaries

Home-based screening for biliary atresia using infant stool colour cards: a large-scale prospective cohort study and cost-effectiveness analysis. Schreiber RA, Masucci L, Kaczorowski J, Collet JP, Lutley P, Espinosa V, Bryan S. J Med Screen. 2014 Sep;21(3):126-32. doi: 10.1177/0969141314542115. Epub 2014 Jul 9.

Purpose: Late diagnosis contributes to poor outcomes for patients with biliary atresia. Researchers looked at the cost-effectiveness of a new screening program designed to help diagnose biliary atresia earlier. Pale stools are a warning sign of biliary atresia.

Methods: Researchers designed a BA Infant Stool Colour Card with information about biliary atresia and pictures of abnormal and normal stool colours. The cards were given to the parents of 6187 healthy babies born at BC Women’s Hospital. Families were asked to check their baby’s stool colour for 30 days following birth and then to mail the cards back to the researchers.  The researchers tested a few different methods to encourage families to use the card, including phone calls and letters. They counted how many families used the cards under each method.

Results and Conclusion: The researchers found that giving the cards to families, without further reminders to use it, was a cost-effective screening strategy for biliary atresia. Since this study was published, a screening program using a stool colour card has started in BC.

Use of corticosteroids after hepatoportoenterostomy for bile drainage in infants with biliary atresia: the START randomized clinical trial. Bezerra JA, Spino C, Magee JC, Shneider BL, Rosenthal P, Wang KS, Erlichman J, Haber B, Hertel PM, Karpen SJ, Kerkar N, Loomes KM, Molleston JP, Murray KF, Romero R, Schwarz KB, Shepherd R, Suchy FJ, Turmelle YP, Whitington PF, Moore J, Sherker AH, Robuck PR, Sokol RJ; Childhood Liver Disease Research and Education Network (ChiLDREN). JAMA. 2014 May 7;311(17):1750-9. doi: 10.1001/jama.2014.2623.

Background: Some doctors give their biliary atresia patients corticosteroids after their Kasai procedure, as it is thought to improve bile flow out of the liver. However, evidence is lacking that supports the effectiveness of corticosteroids for this purpose.

Objective: The researchers wanted to find out whether corticosteroids improved outcomes after the Kasai procedure for biliary atresia patients.

Design: The clinical trial took place at 14 sites between 2005-2011 and included 140 patients. Patients were last followed up in 2013. Patients were divided into two groups: one group received a placebo (ie. no steroids) within 72 hours of the Kasai procedure and the other group received steroids within 72 hours of the Kasai procedure. The group that received steroids received two weeks of IV methylprednisolone followed by two weeks of oral prednisolone, followed by 9 weeks in which the prednisolone dose was gradually decreased.

Results: At 6 months after their Kasai procedures, there was no statistically significant difference in bile drainage between the two groups, although there was a non-statistically significant improvement for the group that received steroids. Transplant-free survival rates were very similar at 2 years of age for both groups. Serious complication rates were very similar between the two groups as well; however, participants in the steroid group on average had their first serious complication 30 days earlier than the non-steroid group.

Conclusion: Steroid therapy did not result in statistically significant bile drainage improvements; however, there was a non-statistically significant improvement indicating that there may be some clinical benefit to receiving steroids. Steroids were associated with earlier onset of serious complications. Based on their results, the researchers do not recommend high-dose steroids following the Kasai procedure for biliary atresia patients.

The most reliable early predictors of outcome in patients with biliary atresia after Kasai’s operation. Goda T, Kawahara H, Kubota A, Hirano K, Umeda S, Tani G, Ishii T, Tazuke Y, Yoneda A, Etani Y, Ida S. J Pediatr Surg. 2013 Dec;48(12):2373-7. doi: 10.1016/j.jpedsurg.2013.08.009.

Purpose: The researchers wanted to determine which factors best predict how well a patient will do after their Kasai operation.

Methods: The researchers looked at the medical charts of 54 patients with biliary atresia who had had a Kasai operation. They divided the patients into two groups: one group where the patients had either died or needed a liver transplant and a second group where the patients were still alive with their native liver (their own liver). The researchers looked at the results of the following blood tests after the Kasai operation: total bilirubin, direct bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), and gamma-glutamyltransferase (GGT).

Results: Total bilirubin, direct bilirubin, AST, and ALT levels after Kasai were lower in the group of patients that eventually survived with their native lower than in the group of patients that eventually either died or needed a liver transplant.

Conclusion: The researchers found that direct bilirubin and AST levels at 2 months after the Kasai operation best predict the long-term health of biliary atresia patients.

Serial ultrasound findings associated with early liver transplantation after Kasai portoenterostomy in biliary atresia. Jeon TY, Yoo SY, Kim JH, Eo H, Lee SK. Clin Radiol. 2013 Jun;68(6):588-94. doi: 10.1016/j.crad.2012.12.004. Epub 2013 Jan 23.

Purpose: The researchers aimed to understand which ultrasound findings are related to early liver transplantation after the Kasai procedure in biliary atresia patients.

Methods: The researchers compared ultrasounds from two groups of children:

Group 1: children with BA who had an early liver transplant (liver transplant at less than 1 year of age) after their Kasai.

Group 2: children with BA who still had their native liver (their own liver) following their Kasai at 1 year of age.

They looked at ultrasounds performed before the Kasai procedure in both groups. They also looked at follow-up ultrasounds for both groups, which were performed before liver transplant for the early liver transplant group (group 1) and performed 1 year after Kasai for patients in the Kasai only group (group 2).

Results: Hepatic artery diameter was larger in the liver transplant group (group 1) than the Kasai only group (group 2) in both the ultrasounds taken before the Kasai and the follow-up ultrasounds. Portal vein diameters were smaller in the early liver transplant group in the follow-up ultrasounds. Splenomegaly (enlarged spleen) at the follow-up ultrasound was more common in the early liver transplant group.

Conclusion: Large hepatic artery prior to Kasai and enlarged spleen at follow-up ultrasound were linked to early liver transplant after Kasai in BA patients.

Health related quality of life in patients with biliary atresia surviving with their native liver Sundaram SS, Alonso EM, Haber B, Magee JC, Fredericks E, Kamath B, Kerkar N, Rosenthal P, Shepherd R, Limbers C, Varni JW, Robuck P, Sokol RJ; Childhood Liver Disease Research and Education Network (ChiLDREN). J Pediatr. 2013 Oct;163(4):1052-7.e2. doi: 10.1016/j.jpeds.2013.04.037. Epub 2013 Jun 6.

Purpose: The researchers aimed to evaluate the health-related quality of life of biliary atresia patients. They compared quality of life of biliary atresia patients with their native livers versus biliary atresia patients who had had liver transplants versus healthy children.

Methods: Researchers evaluated quality of life using the Pediatric Quality of Live Inventory^TM 4.0, which requires the child and child’s parent to fill out a standardized questionnaire. Patients were aged 2-25 years. The questionnaire was completed by 221 patients with their native livers, 151 post-transplant patients, and 954 healthy children.

Results: Quality of life was similar between patients with their native livers compared to patients that were post-transplant. Patients with their native livers showed poorer quality of life than healthy children, especially in emotional and psychosocial functioning. These findings show areas where the health of biliary atresia patients can be most improved.

The long-term outcome of the Kasai operation in patients with biliary atresia: a systematic review. Bijl EJ, Bharwani KD, Houwen RH, de Man RA. Neth J Med. 2013 May;71(4):170-3.

Background: The Kasai operation and liver transplantation are currently the only effective treatments for biliary atresia. A successful Kasai operation postpones the need for a liver transplant by varying amounts. Studies have reported that between 21-44% of biliary atresia patients survive for at least 20-years without a liver transplant. Since it is uncommon for patients to live for a long time following the Kasai operation without a liver transplant, there is little known about the long-term outcomes of the Kasai operation.

Methods and Results: The researchers compiled the results of 14 different studies that looked at the long-term outcomes of the Kasai procedure. The studies included 162 patients over the age of 20 who still had their native liver. They found that 60.5% of these patients had complications such as cholangitis, portal hypertension, and gastrointestinal bleeding. The other 39.5% were alive without complications.

Conclusion: While it is possible for patients with biliary atresia to live long-term without a liver transplant, the majority of these patients will suffer from liver-related complications.

Assessment of risk of bleeding from esophageal varices during management of biliary atresia in children. Wanty C, Helleputte T, Smets F, Sokal EM, Stephenne X. J Pediatr Gastroenterol Nutr. 2013 May;56(5):537-43. doi: 10.1097/MPG.0b013e318282a22c.

Objectives: The best way to treat esophageal varices (abnormally dilated vessels) in patients with biliary atresia is unclear. Esophageal varices can result in gastrointestinal bleeding. A patient’s risk for bleeding can be predicted by certain warning signs, such as red spots in the esophagus and the size of the varices. Some studies show that it is beneficial to treat the varices before bleeding occurs. Others suggest the complications from the treatment outweigh the benefits. This study aimed to find factors that predict the risk of bleeding in biliary atresia patients.

Methods: The study included 83 patients with biliary atresia from one hospital over a span of 7 years. They looked at laboratory results, ultrasound results, and endoscopy results.

Results: Esophageal varices were found in 80% of the patients and 20% of the patients had gastrointestinal bleeding. The study found three risk factors that predict gastrointestinal bleeding: low fibrinogen levels, high-grade (large) esophageal varices, and red colour signs (red spots in the esophagus). Examining these risk factors could help physicians to decide whether to give patients preventative treatments.

Biliary atresia: the Canadian experience. Schreiber RA, Barker CC, Roberts EA, Martin SR, Alvarez F, Smith L, Butzner JD, Wrobel I, Mack D, Moroz S, Rashid M, Persad R, Levesque D, Brill H, Bruce G, Critch J; Canadian Pediatric Hepatology Research Group.  J Pediatr. 2007 Dec;151(6):659-65, 665.e1. Epub 2007 Oct 22.

Study Design: Researchers studied the medical records of babies diagnosed with biliary atresia born in Canada from 1985-1995. They looked at the medical records of 349 babies.

Results: 89% of the patients had a Kasai operation. 4 years after the Kasai operation, 79% of the patients were alive. 2 years after the Kasai operation, 46% of the patients still had their own livers. 4 years after the Kasai operation, 36% of the patients had their own livers. The study showed that patients whose Kasai operation was performed later had lower survival rates. The middle age at Kasai among the patients was 65 days. 60% of the patients had a liver transplantation. Of the patients who had a Kasai followed by a liver transplant, 88% were alive 4 years after the Kasai operation.

Conclusion: Long-term biliary atresia outcomes are similar in Canada to other countries. The Kasai operation is less effective when it is performed later, so late referral to a liver specialist and late Kasai operation is a current problem in Canada.