https://www.ncbi.nlm.nih.gov/pubmed/29377411
Liver Transpl. 2018 Jan 29. doi: 10.1002/lt.25025. [Epub ahead of print]

van der Doef HPJ, van Rheenen PF, van Rosmalen M, Rogiers X, Verkade HJ; pediatric liver transplantation centers of Eurotransplant.

Abstract

INTRODUCTION:
Liver transplantation (LTx) is the standard treatment for biliary atresia (BA) patients with end-stage liver disease. The prognosis after LTx has steadily improved, but overall prognosis of BA-patients is also determined by mortality before LTx. We aimed to quantify mortality in young BA-patients on the Eurotransplant waiting list and to determine the effect of disease severity and age at time of listing on pretransplant mortality.

METHODS:
We used a cohort study design, which incorporated data from the Eurotransplant registry. Participants were 711 BA-patients below 5 years from 5 countries and listed for LTx between 2001-2014. We applied competing risk analysis to evaluate simultaneously the outcomes death, LTx and still-waiting-for-a-suitable-organ. We used Cox proportional hazards regression to assess two-year mortality. In a sub-cohort of 416 children, we performed multivariable analyses between two-year mortality and disease severity or age, each at listing. Disease severity at listing was quantified by the Model for End-stage Liver Disease (MELD) score, which assesses bilirubin, creatinin, albumin and INR as continuous variables.

RESULTS:
Two-years waiting list mortality was 7.9%. Age below 6 months and MELD score above 20 points, each at listing, were strongly and independently associated with two year mortality (each p 0.001). Twenty-one per cent of infants who fulfilled both criteria did not survive the first 6 months on the waiting list.

CONCLUSION:
Our findings quantify mortality among young BA-patients on the waiting list and the relative importance of risk factors (age and severity of disease at listing). Our results provide both an evidence base to rationally address high mortality in subgroups and a methodology to assess effects of implemented changes, for example in allocation rules.