Wait-List Mortality of Young Patients
With Biliary Atresia: Competing Risk
Analysis of a Eurotransplant Registry–
Hubert P. J. van der Doef,
Patrick F. van Rheenen,
Marieke van Rosmalen,
and Henkjan J. Verkade,
for pediatric liver transplantation centers of Eurotransplant
Department of Pediatric Gastroenterology Hepatology and Nutrition, University Medical Center Groningen,
University of Groningen, Groningen, the Netherlands; and
Eurotransplant, Leiden, the Netherlands
Liver transplantation (LT) is the standard treatment for biliary atresia (BA) patients with end-stage liver disease. The
prognosis after LT has steadily improved, but overall prognosis of BA patients is also determined by mortality before LT.
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. We used a cohort study design, which incorporated
data from the Eurotransplant registry. Participants were 711 BA patients who were below 5 years of age from 5 countries
and listed for LT between 2001 and 2014. We applied a competing risk analysis to evaluate simultaneously the outcomes
death, LT, and still waiting for a suitable organ. We used Cox proportional hazards regression to assess 2-year mortality.
In a subcohort of 416 children, we performed multivariate analyses between 2-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, creatinine, albumin, and international normalized ratio as continuous variables. Two-year wait-list mor-
tality was 7.9%. Age below 6 months and MELD score above 20 points, each at listing, were strongly and independently
associated with 2-year mortality (each P < 0.001). A total of 21% of infants who fulﬁlled both criteria did not survive the
ﬁrst 6 months on the waiting list. In 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.
Liver Transplantation 24 810–819 2018 AASLD.
Received September 19, 2017; accepted January 17, 2018.
SEE EDITORIAL ON PAGE 731
Biliary atresia (BA) is an obstructive cholangiopathy
that usually arises in the perinatal period. The surgical
development of the Kasai portoenterostomy, which
aims to restore bile ﬂow from liver to intestine, has
greatly improved the prognosis of infants with BA.
However, approximately 50% of those who undergo
Kasai operation still develop end-stage liver disease
before the age of 5 years and require liver transplanta-
BA is the most common indication for
LT in the pediatric age group.
Nowadays, 1-year survival after pediatric LT is close
However, the overall prognosis of BA
patients is greatly affected by mortality before trans-
Eurotransplant is a nonproﬁt organiza-
tion that facilitates the patient-oriented allocation in
8 Western and central European countries and the
cross-border exchange of deceased donor liver trans-
plantation (DDLT) organs. The organization has a
multicenter registry that covers all patients listed for
Abbreviations: BA, biliary atresia; CI, conﬁdence interval; DDLT,
deceased donor liver transplantation; ELIAC, Eurotransplant Liver
Intestine Advisory Committee; INR, international normalized ratio;
LDLT, living donor liver transplantation; LT, liver transplanta-
tion; MELD, Model for End-Stage Liver Disease; PELD, Pediatric
End-Stage Liver Disease.
VAN DER DOEF ET AL.