Original Article
Biliary Atresia: Clinical Profiles, Risk Factors, and Outcomes of 755 Patients Listed for Liver Transplantation

https://doi.org/10.1016/j.jpeds.2005.04.073Get rights and content

Objectives

To test the hypothesis that risk analysis from the time of listing for liver transplantation (LT) focuses attention on areas where outcomes can be improved.

Study design

Competing outcomes and multivariate models were used to determine significant risk factors for pretransplantation and posttransplantation mortality and graft failure in patients with biliary atresia (BA) listed for LT and enrolled in the Studies of Pediatric Liver Transplantation (SPLIT) registry.

Results

Of 755 patients, most were infants (age < 1 year). Significant waiting list mortality risk factors included infancy and pediatric end-stage liver disease (PELD) score ≥ 20, whose components were also continuous risk factors. Survival posttransplantation (n = 567) was 88% at 3 years. Most deaths were from infection (37%). Posttransplantation mortality risk factors included infant recipients, height/weight < −2 standard deviations (SD), use of cyclosporine versus tacrolimus and retransplantation. Graft failure risks included height/weight < −2 SD, cadaveric partial donors, donor age ≤ 5 months, use of cyclosporine versus tacrolimus, and rejection.

Conclusions

Referral for LT should be anticipatory for infants with BA with failed portoenterostomies. Failing nutrition should prompt aggressive support. Post-LT risk factors are mainly nonsurgical, including nutrition, the relative risk of infection over rejection, and the choice of immunosuppression.

Section snippets

Patient Population

The study group comprised all 755 children < age 18 years with BA listed for their first LT and enrolled in the SPLIT registry from May 1995 to June 2003. As described previously, all of the 39 SPLIT centers had Institutional Review Board approval or a waiver for data collection and analysis.14, 15, 16 Individual informed consent was obtained from parents and/or guardians. Coded information was submitted to the SPLIT data-coordination center at the time of listing for LT. Follow-up data were

Characteristics of Patients With BA Listed for LT

Clinical and demographic details of the 755 patients with BA at the time of listing for LT are summarized in Table I (available online at www.mosby.com/jpeds). More than 70% of the patients were < 1 year of age, and 60% were female. Most (82%) were not hospitalized at the time of listing. Most had PELD scores between 10 and 20 (mean, 11.7; median, 12.1). More than 40% of patients had growth failure, although only 16% received nasogastric supplements. The mean height z-score at listing was −1.3

Discussion

These data, the largest cumulative dataset available to date describing outcomes from the time of listing for LT in patients with BA needing such therapy, reflect the current state of the art in the United States and Canada. Special considerations regarding LT for BA apply, including the predominance of infant recipients, difficulties in predicting outcomes, and timing of donor acquisition. To date, clinical profiles, likelihood of living or dying while on the LT waiting list, and donor

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  • Cited by (0)

    SPLIT (Studies of Pediatric Liver Transplantation) is a consortium of 39 pediatric liver transplant centers in the United States and Canada (http://www.splitregistry.com). SPLIT is supported by a grant (1 U01 DK061693-01A1) from the National Institute of Diabetes and Digestive and Kidney Diseases, an unrestricted educational grant from Fujisawa Healthcare, and additional support from Wyeth-Ayerst Labs, Roche Labs, Medimmune Inc., and Novartis.

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