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).
Pediatric liver transplantation for urea cycle disorders and organic acidemias: United Network for Organ Sharing data for 2002-2012
Article first published online: 29 NOV 2013
© 2013 American Association for the Study of Liver Diseases
Volume 20, Issue 1, pages 89–99, January 2014
How to Cite
Perito, E. R., Rhee, S., Roberts, J. P. and Rosenthal, P. (2014), Pediatric liver transplantation for urea cycle disorders and organic acidemias: United Network for Organ Sharing data for 2002-2012. Liver Transpl, 20: 89–99. doi: 10.1002/lt.23765
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
- Issue published online: 20 DEC 2013
- Article first published online: 29 NOV 2013
- Accepted manuscript online: 17 OCT 2013 01:30PM EST
- Manuscript Accepted: 30 SEP 2013
- Manuscript Received: 11 JUL 2013
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.