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Liver transplantation for urea cycle disorders in pediatric patients: A single‐center experience
Author(s) -
Kim Irene K.,
Niemi AnnaKaisa,
Krueger Casey,
Bonham Clark A.,
Concepcion Waldo,
Cowan Tina M.,
Enns Gregory M.,
Esquivel Carlos O.
Publication year - 2013
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12041
Subject(s) - medicine , neurocognitive , liver transplantation , hyperammonemia , transplantation , biliary atresia , single center , pediatrics , urea cycle , retrospective cohort study , population , surgery , cognition , psychiatry , biochemistry , chemistry , environmental health , amino acid , arginine
LT has emerged as a surgical treatment for UCD s. We hypothesize that LT can be safely and broadly utilized in the pediatric population to effectively prevent hyperammonemic crises and potentially improve neurocognitive outcomes. To determine the long‐term outcomes of LT for UCD s, charts of children with UCD who underwent LT were retrospectively reviewed at an academic institution between J uly 2001 and M ay 2012. A total of 23 patients with UCD underwent LT at a mean age of 3.4 yr. Fifteen (65%) patients received a whole‐liver graft, seven patients (30%) received a reduced‐size graft, and one patient received a living donor graft. Mean five‐yr patient survival was 100%, and allograft survival was 96%. Mean peak blood ammonia ( NH 3 ) at presentation was 772 μmol/L (median 500, range 178–2969, normal <30–50). After transplantation, there were no episodes of hyperammonemia. Eleven patients were diagnosed with some degree of developmental delay before transplantation, which remained stable or improved after transplantation. Patients without developmental delay before transplantation maintained their cognitive abilities at long‐term follow‐up. LT was associated with the eradication of hyperammonemia, removal of dietary restrictions, and potentially improved neurocognitive development. Long‐term follow‐up is underway to evaluate whether LT at an early age (<1 yr) will attain improved neurodevelopmental outcomes.