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Original Investigation
November 2018

Accuracy of the Pediatric End-stage Liver Disease Score in Estimating Pretransplant Mortality Among Pediatric Liver Transplant Candidates

Author Affiliations
  • 1Department of Medicine, School of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania
  • 2Department of Biostatistics, Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania
  • 3Department of Clinical and Translational Science, School of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania
  • 4Department of Health Policy and Management, Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania
  • 5Department of Pediatrics, Baylor College of Medicine, Houston, Texas
  • 6Department of Industrial Engineering, Swanson School of Engineering, University of Pittsburgh, Pittsburgh, Pennsylvania
  • 7Department of Pediatrics, School of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania
JAMA Pediatr. 2018;172(11):1070-1077. doi:10.1001/jamapediatrics.2018.2541
Key Points

Question  Do Pediatric End-stage Liver Disease scores adequately estimate 90-day pretransplant mortality among pediatric patients?

Findings  In this study of 4298 patients with chronic liver disease identified using the United Network for Organ Sharing pediatric waiting list, Pediatric End-stage Liver Disease scores and mortality were concordant; however, the estimated risk using the Pediatric End-stage Liver Disease score significantly underestimated actual 90-day mortality.

Meaning  The Pediatric End-stage Liver Disease system may be flawed and may disadvantage children awaiting liver transplant when used to adjudicate organ allocation decisions; a new system that reflects actual 90-day mortality should be developed.

Abstract

Importance  Fair allocation of livers between pediatric and adult recipients is critically dependent on the accuracy of mortality estimates afforded by the Pediatric End-stage Liver Disease (PELD) and Model for End-stage Liver Disease, respectively. Widespread reliance on exceptions for pediatric recipients suggests that the 2 systems may not be comparable.

Objective  To evaluate the accuracy of the PELD score in estimating 90-day pretransplant mortality among pediatric patients on the United Network for Organ Sharing (UNOS) waiting list.

Design, Setting, and Participants  Patients who were listed from February 27, 2002, to March 31, 2014, for primary liver transplant were included in this retrospective analysis and were followed up for at least 2 years through June 17, 2016. The study analyzed 2 cohorts using the UNOS Standard Transplant Analysis and Research data files. The full cohort comprised 4298 patients (<18 years of age) who had chronic liver disease (excluding cancer). The reduced cohort (n = 2421) excluded patients receiving living donor transplantation or PELD exception points.

Main Outcomes and Measures  Observed and expected 90-day pretransplant mortality rates evaluated at 10-point interval PELD levels.

Results  Among the 4298 patients in the full cohort (mean [SD] age, 2.5 [4.2] years; 2251 [52.4%] female; 2201 [51.2%] white), PELD scores and mortality were concordant (C statistic, 0.8387 [95% CI, 0.8191-0.8584] for the full cohort and 0.8123 [95% CI, 0.7919-0.8327] for the reduced cohort). However, the estimated 90-day mortality using the PELD score underestimated the actual probability of death by as much as 17%.

Conclusions and Relevance  With use of the PELD score, the ranking of risk among children was preserved, but direct comparisons between adult and pediatric candidates were not accurate. Children with chronic liver disease who are in need of transplant may be at a disadvantage compared with adults in a similar situation.

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