Impaired intention‐to‐treat survival after listing for liver transplantation in children with biliary atresia compared to other chronic liver diseases: 20 years’ experience from the Nordic countries

Abstract

Biliary atresia (BA) is the most common indication for LT in children. We investigated whether this diagnosis per se, compared to other chronic liver diseases (OCLD), had an influence on patient survival. Data from 421 Scandinavian children, 194 with BA and 227 with OCLD, listed for LT between 1990 and 2010 were analyzed. The intention-to-treat survival and influencing risk factors were studied. Patients with BA had higher risk of death after listing than patients with OCLD. The youngest (<1 year) and smallest (<10 kg) children with the highest bilirubin (>510 μmol/L), highest INR (>1.6), and highest PELD score (>20) listed during 1990s had the worst outcome. Given the same PELD score, patients with BA had higher risk of death than patients with OCLD. For adolescents, low weight/BMI was the only prognostic marker. Impaired intention-to-treat survival in patients with BA was mainly explained by more advanced liver disease in younger ages and higher proportion of young children in the BA group rather than diagnosis per se. PELD score predicted death, but seemed to underestimate the severity of liver disease in patients with BA. Poor nutritional status and severe cholestasis had negative impact on survival, supporting the "sickest children first" allocation policy and correction of malnutrition before surgery.

A paradigm shift in the intention‐to‐transplant children with biliary atresia: Outcomes of 101 cases and a review of the literature

“…This suggests that a progressing liver dysfunction (thus PELD) is more relevant than malnutrition per se and that children who deteriorate their liver function rapidly should be referred to transplant centers independently of their age and weight. This is in line with a series of recent reports that have highlighted that the risk of death, while waiting for a transplant, was directly related to (a) a relatively late referral to the transplant team, even though these infants were young (<1 year of age at referral); and (b) a poor clinical condition (typically a combination of small weight and malnutrition, severe cholestasis, and a rapidly progressing hepatic dysfunction). Both of these aspects are directly related to each other and represent a failure of our current referral systems; because the failure of a Kasai procedure can be recognized within 2‐3 months of the procedure, and because it is per se an indication for LT, there is little that can explain why children would be referred late or in poor condition.…”

“…An early and rapid failure after Kasai operation is in fact not a rare condition. Worse, it is associated with a high death rate on waiting list in at least three recent reports from three large networks: Eurotransplant (ET), UNOS, and the Nordic countries . The former group of children must be identified early, with the objective of a very early referral.…”

“…Worse, it is associated with a high death rate on waiting list in at least three recent reports from three large networks: Eurotransplant (ET), UNOS, and the Nordic countries. [7][8][9] The former group of children must be identified early, with the objective of a very early referral. In order to do so, it will be necessary to define a set of criteria that will allow for identifying that condition.…”

“…The period of the analyses was from 2003 to 2013, 2001‐2014, 2004‐2010, and 1997‐2008, respectively. The death rates of BA patients on these waiting lists were 12.4% at <2 years of age, 7.9% at <5 years of age, 19% at >2 years of age, and 16% at >5 years of age, respectively …”

Wait‐list mortality of young patients with Biliary atresia: Competing risk analysis of a eurotransplant registry–based cohort

“…We applied the competing risk method because this analyzes risk of death on the waiting list while accounting for nondeath events (such as transplantation). Among 4 articles on wait‐list mortality in children with BA, only 1 may have used the competing risk analysis, but the competing risks were not shown over time in 1 graph.…”