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Purpose: The purpose of this study was to investigate factors impacting transplant-free survival among infants with biliary atresia. Methods: A multi-institutional, retrospective cohort study was performed at nine tertiary-level children's hospitals in the United States. Infants who underwent Kasai portoenterostomy (KP) from January 2009 to May 2017 were identified. Clinical characteristics included age at time of KP, steroid use, surgical approach, liver pathology, and surgeon experience. Likelihood of transplant-free survival (TFS) was evaluated using logistic regression, adjusting for patient and surgeon-level factors. Secondary outcomes at 1 year included readmission, cholangitis, reoperation, mortality, and biliary clearance. Results: Overall, 223 infants underwent KP, and 91 (40.8%) survived with their native liver. Mean age at surgery was 63.9 days (± 24.7 days). At 1 year, 78.5% experienced readmission, 56.9% developed cholangitis, 3.8% had a surgical revision, and 5 died. Biliary clearance at 3 months was achieved in 76.6%. Controlling for patient and surgeon-level factors, each additional day of age toward operation was associated with a 2% decrease in likelihood of TFS (OR 0.98, 95% CI 0.97–0.99). Conclusion: Earlier surgical intervention by Kasai portoenterostomy at tertiary-level centers significantly increases likelihood for TFS. Policy-level interventions to facilitate early screening and surgical referral for infants with biliary atresia are warranted to improve outcomes. [ABSTRACT FROM AUTHOR]

Objective: To investigate the epidemiological data, prognostic factors, and treatment outcomes of bone angiosarcoma (BA). Methods: This retrospective study was based on the Surveillance, Epidemiology, and End Results (SEER) database. The medical records of BA patients were selected from the SEER database from 1975 to 2016. Variables including patients' baseline demographics (age, sex, marital status, and year of diagnosis), tumor characteristics (tumor size, grade, and SEER Historic Stage A), and treatment (surgery and radiotherapy) were selected for further analysis. The research endpoints were overall survival (OS) and cancer‐specific survival (CSS). The optimal cutoff values of continuous variables including age, year of diagnosis, and tumor size were identified using the X‐tail program. Univariate Cox regression was used to identify potential prognostic factors and multivariate Cox regression was used to identify independent prognostic factors. All prognostic factors were included to predict the survival time compared to the median OS and CSS times via the novel nomograms. To validate the internal validation of nomograms, we analyzed the concordance indices (C‐index). Results: This study enrolled a total of 271 patients with malignant vascular bone tumors among residents of the United States between 1975 and 2016. After applying the exclusion criteria (one case without active follow‐up), this study included 152 patients with BA. The median survival time of BA was significantly shorter than that of malignant vascular bone tumors for OS (9 months vs 27 months, P < 0.001). Age, year of diagnosis, tumor size, grade, stage, and surgery were identified as potential prognostic factors for OS or CSS in univariate Cox regression. However, only age (P < 0.001, P < 0.001), stage (P = 0.002, P < 0.001), and surgery (P = 0.001, P = 0.002) were independent prognostic factors for CSS and OS, respectively, in the multivariate analysis. Younger patients less than 54 years have significantly better prognosis for CSS/OS than patients between 54 and 67 years (Hazard ratios [HRs]: 1.651 [1.763–3.575], 2.557 [1.395–4.687]) and more than 67 years (HRs: 4.404 [2.237–8.670], 5.113 [2.923–8.942]). For CSS/OS, the survival time of patients with localized stage was significantly longer than that of patients with regional stage (HRs: 1.530 [0.725–3.228], 1.548 [0.834–2.873]) and that of patients with distant stage (HRs: 1.706 [0.899–3.237], 2.101 [1.254–3.520]). Patients with surgery had more survival time than patients without surgery for CSS/OS (HRs: 2.861 [1.542–5.310], 2.103 [1.308–3.379]). All factors were further included to generate nomograms for CSS and OS. The C‐indexes for the internal validation of OS and CSS prediction were 0.787 (95% confidence interval [CI]: 0.738–0.836) and 0.768 (95% CI: 0.717–0.819), respectively. Conclusions: Age, stage, and surgery were closely associated with prognosis in patients with BA, and this clinical model was a favorable tool to evaluate survival possibilities. [ABSTRACT FROM AUTHOR]

Background: Biliary atresia (BA) remains the number one indication for paediatric liver transplantation (LT) worldwide but is an uncommon indication for older LT recipients. The impact of recent donor allocation changes, pervasive organ shortage and evolving LT practices on the BA LT population is unknown., Methods: We identified patients who underwent LT between January 2010 and December 2021 using the UNOS database. We compared clinical outcomes between patients with BA and those with non-BA cholestatic liver disease. Groups were stratified by age, <12 years (allocated via PELD system) and ≥12 years (allocated via MELD system). Waitlist outcomes were compared using competing-risk regression analysis, graft survival rates were compared using Kaplan-Meier time-to-event analysis and Cox proportional hazards modelling provided adjusted estimates., Results: There were 2754 BA LT waitlist additions and 2206 BA LTs (1937 <12 years [younger], 269 ≥12 years [older]). There were no differences in waitlist mortality between BA and non-BA cholestatic patients. Among BA LT recipients, there were 441 (20.0%) living-donor liver transplantations (LDLT) and 611 (27.7%) split deceased-donor LTs. Five-year graft survival was significantly higher among BA versus non-BA cholestatic patients in the older group (88.3% vs. 79.5%, p < .01) but not younger group (89.3% vs. 89.5%). Among BA LT recipients, improved graft outcomes were associated with LDLT (vs. split LT: HR: 2, 95% CI: 1.03-3.91) and higher transplant volume (volume >100 vs. <40 BA LTs: HR: 3.41, 95% CI: 1.87-6.2)., Conclusion: Liver transplant outcomes among BA patients are excellent, with LDLT and higher transplant centre volume associated with optimal graft outcomes., (© 2023 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)

Despite advances in our understanding of the pathogenesis of biliary atresia (BA), BA remains the most common cause of end-stage liver disease in children and the leading indication for pediatric liver transplantation. Age at time of Kasai portoenterostomy (KPE), performed to provide bile drainage, strongly correlates with transplant-free survival, mostly due to progression of intrahepatic fibrosis to cirrhosis. Unfortunately, challenges remain in recognizing that a jaundiced infant may have BA. To better diagnose infants with BA at an earlier age, population-based screening programs in countries such as Taiwan, Japan, and China have utilized stool color cards. Early results have been promising demonstrating earlier diagnosis, earlier KPE, and, hence, improved outcomes. Cost-effectiveness studies focused on stool color card screening in North America where the incidence of BA is much lower also project improved transplant-free survival rate with a savings in terms of healthcare expenditure. There is also evidence that postnatal serum bilirubin levels may also be effective as a screening tool given that all infants with BA exhibit hyperbilirubinemia at birth. The American Academy of Pediatrics (AAP) recently advocated studying the implementation of newborn screening for BA in the United States. Further efforts and analyses within the United States are ongoing, but current evidence is supportive of screening for BA even in low incidence countries. [ABSTRACT FROM AUTHOR]

Background: Living donor liver transplants (LDLTs) including those from nondirected donors (NDDs) have increased during the past decade, and center-level variations in LDLTs have not yet been described. We sought to quantify changes in the volume of NDD transplants over time and variation in NDD volume between transplant centers. We further examined characteristics of living liver donors and identified factors potentially associated with receiving an NDD liver transplant., Methods: Using Scientific Registry of Transplant Recipients data between March 01, 2002, and December 31, 2020, we compared 173 NDDs with 5704 DLDs and 167 NDD recipients with 1153 waitlist candidates., Results: NDDs increased from 1 (0.4% of LDLTs) in 2002 to 58 (12% of LDLTs) in 2020. Of 150 transplant centers, 35 performed at least 1 NDD transplant. Compared with waitlist candidates, adult NDD recipients were less frequently males (39% versus 62%, P < 0.001), had a lower model for end-stage liver disease (16 versus 18, P  = 0.01), and spent fewer days on the waitlist (173 versus 246, P  = 0.02). Compared with waitlist candidates, pediatric NDD recipients were younger (50% versus 12% age <2 y, P < 0.001) and more often diagnosed with biliary atresia (66% versus 41%, P < 0.001). Compared with DLDs, NDDs were older (40 versus 35 y, P < 0.001), college educated (83% versus 64%, P < 0.001), White (92% versus 78%, P < 0.001), and more frequently donated left-lateral segment grafts (32.0% versus 14%, P < 0.001)., Conclusions: Liver NDD transplants continue to expand but remain concentrated at a few centers. Graft distribution favors female adults and pediatric patients with biliary atresia. Racial inequities in adult or pediatric center-level NDD graft distribution were not observed., Competing Interests: The authors declare no conflicts of interest., (Copyright © 2022 Wolters Kluwer Health, Inc. All rights reserved.)

Karnsakul W, Intihar P, Konewko R, Roy A, Colombani PM, Lau H, Schwarz KB. Living donor liver transplantation in children: A single North American center experience over two decades. Abstract: Little data concerning hospital charges and long-term outcomes of LDLT in North American children according to transplant indications have been published. To compare outcomes of patient and graft survival and healthcare charges for LDLT for those with BA vs. other diagnoses (non-BA). A retrospective review of 52 children receiving 53 LDLT (38 BA and 14 non-BA) from 1992 to 2010 at our institution was performed. One-, five-, and 10-yr patient and graft survival data were comparable to national figures reported to UNOS. Average one-yr charges for recipients and donors were $242 849 for BA patients and $183 614 for non-BA (p = 0.074). BA patients were 1.23 ± 1.20 yr of age vs. 4.25 ± 5.02 for non-BA, p = 0.045. Examination of the total population of patients who were alive in 2010 in five chronological groupings showed that the crude five-yr survival rates were 1992-1995: 9/11 (82%); 1995-1997: 6/10 (60%); 1997-1999: 8/10 (80%); 1999-2001: 9/10 (90%); and 2001-2003: 7/7 (100%). Thus, examination of the clinical and financial data together over the entire period of the transplant program suggests that the dramatic improvement in patient survival was accomplished without a dramatic increase in indexed charges. All 53 donors survived, and only 10% had complications requiring hospitalization. LDLT in children results in excellent outcomes for patients and donors. Ways to lower costs and maximize graft outcome should be investigated. [ABSTRACT FROM AUTHOR]

Background: Biliary atresia is a rare but devastating disease for which hepatoportoenterostomy remains the primary intervention. Increased age at the time of hepatoportoenterostomy is associated with unfavorable outcomes. In this study, we examined trends in age at the time of hepatoportoenterostomy and explored hospital and patient factors associated with more timely diagnosis and treatment. Methods: Median ages of patients undergoing hepatoportoenterostomy for biliary atresia were compared using the Kids'' Inpatients Database from 1997, 2000, 2003, and 2006. The patient and hospital factors associated with later treatment were compared. Results: Of 192 patients, 13.5% had surgery in 1997, 13.5% in 2000, 36.5% in 2003, and 36.5% in 2006. The overall median age was 65.5 days; the median age was 64 days in 1997, 57.5 days in 2000, 69 days in 2003, and 64 days in 2006 (P = .80). Overall, 71% of patients were treated at nonchildren''s hospitals, and although the proportion has increased over time, the trend did not reach significance (P = .12). Hispanic and African American patients were more likely to undergo hepatoportoenterostomy after 60 days of life compared with white patients (Hispanic patients: odds ratio, 3.6; 95% confidence interval, 1.1–12.5; P = .04; African American patients: odds ratio, 2.2; 95% confidence interval, 0.8–6.3; P = .14). Compared with specialized children''s centers, treatment at nonchildren''s hospitals was associated with delayed hepatoportoenterostomy (odds ratio, 3.5; 95% confidence interval, 1.2–9.8; P = .02). Conclusion: Although early hepatoportoenterostomy is associated with improved outcomes for children with biliary atresia, our study shows the median age at surgery has not significantly changed over 2 decades. Both hospital and socioeconomic factors play a role in the early treatment of biliary atresia. [Copyright &y& Elsevier]

During the last decade, biliary atresia (BA) has attained more interest and the frequency of BA-related publications has increased continuously. Pediatric hepatologists and pediatric surgeons are very active in improving diagnosis and treatment modalities of BA patients, in order to prolong the survival rate of their native liver. Together with transplant surgeons, the bridging of BA patients to liver transplantation (LTx) becomes optimized and as a consequence of this interdisciplinary approach, the overall survival of babies with BA has already reached 90%. Furthermore, basic research into the still unknown origin of BA has advanced, and numerous scientific programs have already linked together. The overriding interest is to discover at least BA's etiology and to turn the treatment of BA patients from a symptomatic to a causative approach. Interdisciplinary and international programs are mandatory and already existing initiatives in Europe, the United States and Japan are going to coordinate their registries, clinical trials and basic research studies for the benefit of the patients and solve the riddle of BA. [ABSTRACT FROM AUTHOR]

The aetiology of biliary atresia, the leading cause of neonatal extrahepatic jaundice and the main indication for liver transplantation in children, is unknown. Recent research has focused on an infectious aetiology and the development of viral models in animals. The few published epidemiological studies report conflicting results for seasonal, geographical, and racial variations in incidence. In this study, New York State (NYS) Congenital Malformations Registry data from 1983 to 1998 were compared with resident live birth certificate data. County of residence, birth date, gestational age, birthweight, gender, maternal race and maternal age were extracted from the birth certificate data. Isolated and sequence cases were combined for analysis. Observed and expected numbers of cases were calculated by NYS region. Overall, 369 biliary atresia cases were reported in the 16-year study period, a rate of 0.85 [95% CI 0.76, 0.93] per 10 000 live births. Of these, 249 isolated/sequence cases were ascertained, a rate of 0.57 [95% CI 0.50, 0.64] per 10 000 live births. The rate ratio of biliary atresia in New York City (NYC) compared with other NYS was 2.19 [95% CI 1.69, 2.84]. Seasonal patterns varied by region with spring births at highest risk in NYC and September to November births at highest risk in other NYS. The rate ratio in black vs. white mothers was 1.94 [95% CI 1.48, 2.54]. Birthweight and gestational age were associated with biliary atresia with preterm low-birthweight infants at highest risk [RR 3.24, 95% CI 2.20, 4.76]. The association of isolated/sequence biliary atresia with season, preterm birth, and low birthweight in our study supports an infectious disease hypothesis. [ABSTRACT FROM AUTHOR]

Abstract Liver diseases which affect children are unique. Diseases such as inborn errors of metabolism are fertile grounds for investigation. Study of these ‘experiments of nature’ will provide insight into hepatobiliary function and lead to novel treatment modalities. Successful treatment or prevention of liver disease in children will have long-term implications. This report describes the current burden of pediatric liver disease and offers approaches to intervention. © 2002 Blackwell Publishing Asia Pty Ltd. [ABSTRACT FROM AUTHOR]

Analyzes the temporal and demographic distribution of biliary atresia using a population-based birth defect surveillance system in metropolitan Atlanta, Georgia. Result of the mapping of the location of cases; Prevalence rate of the disease; Evidences of seasonal variation; Significant factors in the development of biliary atresia.

We describe a 5-week-old infant who presented in cardiac arrest and was later found to have intracranial hemorrhage due to late vitamin K deficiency bleeding. The infant had not received vitamin K prophylaxis following a home birth. Because of worsening direct hyperbilirubinemia and transaminitis, she was subsequently found to have biliary atresia. This case allows us to review the differential diagnosis of intracranial hemorrhage in infants and underscores the importance of considering whether infants presenting with any source of bleeding had received vitamin K prophylaxis. As refusal of vitamin K prophylaxis has increased in the United States over the last decade, pediatricians now play an increasingly vital role in promoting strong adherence to universal prophylaxis for newborns in their practice. [ABSTRACT FROM AUTHOR]

Background: Kasai portoenterostomy (KPE) remains the first-line operation for patients with biliary atresia (BA), but ultimately fails in up to 60% of cases. This study sought to identify factors contributing to hospital readmission and early liver transplant., Methods: The Nationwide Readmissions Database from 2010 to 2014 was used to identify patients with BA who underwent KPE on index admission. Patient factors, hospital characteristics, and complications of BA were compared by readmission rates and rate of liver transplant within 1 y. The results were weighted for national estimates., Results: Nine hundred and sixty three patients were identified. The readmission rate within 30-d was 36% (n = 346) and within 1-y was 67% (n = 647). Only 9% (n = 90) received a liver transplant within a year. The most common complications after KPE were cholangitis in 58%, decompensated cirrhosis in 54%, and recurrent jaundice in 34%. Male patients (OR 1.5, P = 0.02) with comorbid gastrointestinal anomalies (OR 2.1, P < 0.01) from lower income households (OR 4.6, P < 0.01) and early development of cirrhosis (OR 3.0, P < 0.01) were more likely to be readmitted. Liver transplant was more common in men (OR 4.0, P < 0.01) and those from lower income households (OR 5.2, P < 0.01) with decompensated cirrhosis (OR 8.6, P < 0.01), cholangitis (OR 5.0, P < 0.01), or sepsis (OR 5.7, P < 0.01) on index admission., Conclusions: This is the first nationwide study to evaluate readmissions in patients with BA undergoing KPE. Although KPE is a lifesaving procedure, hospital readmission rates are high and complications are common. Cholangitis, early progression of cirrhosis, and infections are highly associated with readmission and failure of KPE., (Copyright © 2020 Elsevier Inc. All rights reserved.)

In areas of the world where human herpesvirus 8 (HHV‐8) is endemic, Kaposi sarcoma (KS) is a common SOT‐associated cancer. In the United States, where the virus is not prevalent, PTKS is rare, and there is little literature on pediatric PTKS. We present a North American female who underwent deceased donor, left lateral segment liver transplant for biliary atresia at age 11 months. The donor was a male with no known history of KS, originally from an HHV‐8‐endemic country. Three months after transplantation, the patient developed liver nodules and portal vein thrombosis. Analysis of needle biopsy established the diagnosis of KS and confirmed that the transformed cells were donor‐derived. HHV‐8 viremia was detected, and ganciclovir dosing (which had been started prophylactically) was increased. Immunosuppression was changed from tacrolimus to sirolimus. After further disease progression, 8 cycles of paclitaxel were administered. Under this treatment, her nodules regressed, HHV‐8 viremia resolved, and she had marked clinic improvement. Notably, the adult recipient of the right liver lobe from the same donor also developed PTKS. This is one of few pediatric PTKS cases described in the literature. It contributes to the mechanistic understanding of PTKS development, illustrating the risk posed by donors from HHV‐8‐endemic countries, as well as the potential for strong PTKS correlation between multiple recipients of organs from a single shared donor. [ABSTRACT FROM AUTHOR]

Investigates the epidemiology of extrahepatic biliary atresia in New York. Determination of relative risk among newborns; Significance of the month of conception; Impact of living in densely populated areas.