In:
Pediatric Transplantation, Wiley, Vol. 21, No. 6 ( 2017-09)
Abstract:
UCD s are among the most common inherited metabolic diseases in Japan. We investigated the clinical manifestations, treatment, and prognoses of 177 patients with UCD s who were evaluated and treated from January 1999 to March 2009 in Japan, using a questionnaire survey. Among these 177 patients, 42 (seven with carbamoyl phosphate synthetase 1 deficiency, 27 with ornithine transcarbamylase deficiency, seven with argininosuccinate synthetase deficiency, and one with arginase 1 deficiency) underwent living‐donor LT . Although this study was retrospective and included limited neurodevelopmental information before and after LT , we evaluated whether LT could improve neurodevelopmental outcomes in patients with UCD s. The neurodevelopmental outcomes of patients with a MAC of 〈 300 μmol/L at the time of onset were not significantly different between the LT and non‐ LT groups ( P =.222). LT may have prevented further neurodevelopmental complications in children with MAC ≥300 μmol/L ( P =.008) compared with non‐transplant management. Therefore, Liver transplant should be considered in patients with UCD with a MAC of ≥300 μmol/L at the time of disease onset.
Type of Medium:
Online Resource
ISSN:
1397-3142
,
1399-3046
DOI:
10.1111/petr.2017.21.issue-6
Language:
English
Publisher:
Wiley
Publication Date:
2017
detail.hit.zdb_id:
2008614-3