Pediatric liver transplantation for urea cycle disorders and organic acidemias: United Network for Organ Sharing data for 2002-2012

Authors

  • Emily R. Perito,

    Corresponding author
    1. Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California San Francisco, San Francisco, CA
    • Address reprint requests to Emily R. Perito, M.D., Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California San Francisco, 500 Parnassus Avenue, MU408E, Box 0136, San Francisco, CA 94143. Telephone: 415-476-5892; FAX: 415-476-1343; E-mail: peritoe@peds.ucsf.edu

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  • Sue Rhee,

    1. Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California San Francisco, San Francisco, CA
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  • John Paul Roberts,

    1. Division of Transplant Surgery, Department of Surgery, University of California San Francisco, San Francisco, CA
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  • Philip Rosenthal

    1. Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of California San Francisco, San Francisco, CA
    2. Division of Transplant Surgery, Department of Surgery, University of California San Francisco, San Francisco, CA
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  • This project was supported by the American Gastroenterological Association through the Emmet B. Keeffe Career Development Award in Clinical or Translational Research in Liver Disease (to Emily R. Perito), by the National Institutes of Health/National Center for Research Resources (grant UL1 RR024131 to the University of California San Francisco Clinical and Translational Science Institute), and by the Health Resources and Services Administration (contract 234-2005-370011C for United Network for Organ Sharing data).

  • The authors have no relevant conflicts of interest to disclose.

  • The content of this article is the responsibility of the authors alone and does not necessarily reflect the views or policies of the American Gastroenterological Association, the National Institutes of Health, or the Department of Health and Human Services, nor does mention of trades names, commercial products, or organizations imply endorsement by the US Government.

  • See Editorial on Page 1

Abstract

Decision making concerning liver transplantation is unique for children with urea cycle disorders (UCDs) and organic acidemias (OAs) because of their immediate high priority on the waiting list, which is not related to the severity of their disease. There are limited national outcome data on which recommendations about liver transplantation for UCDs or OAs can be based. This study was a retrospective analysis of United Network for Organ Sharing data for liver recipients who underwent transplantation at an age < 18 years in 2002-2012. Repeat transplants were excluded. Among the pediatric liver transplants, 5.4% were liver-only for UCDs/OAs. The proportion of transplants for UCDs/OAs increased from 4.3% in 2002-2005 to 7.4% in 2010-2012 (P < 0.001). Ninety-six percent were deceased donor transplants, and 59% of these patients underwent transplantation at <2 years of age. Graft survival improved as the age at transplant increased (P = 0.04). Within 5 years after transplantation, the graft survival rate was 78% for children < 2 years old at transplant and 88% for children ≥ 2 years old at transplant (P = 0.06). Vascular thrombosis caused 44% of the graft losses, and 65% of these losses occurred in children < 2 years old. Patient survival also improved as the age at transplant increased: the 5-year patient survival rate was 88% for children with UCDs/OAs who were <2 years old at transplant and 99% for children who were ≥2 years old at transplant (P = 0.006). At the last-follow-up (54 ± 34.4 months), children who underwent transplantation for UCDs/OAs were more likely to have cognitive and motor delays than children who underwent transplantation for other indications. Cognitive and motor delays for children with UCDs/OAs were associated with metabolic disorders, but they were not predicted by age or weight at transplant, sex, ethnicity, liver graft type (split versus whole), or hospitalization at transplant in univariate and multivariate analyses. In conclusion, most liver transplants for UCDs/OAs occur in early childhood. Further research on the benefits of early transplantation for patients with UCDs/OAs is needed because a younger age may increase posttransplant morbidity. Liver Transpl 20:89-99, 2014. © 2013 AASLD.

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