Treatment of ornithine transcarbamylase deficiency in girls by auxiliary liver transplantation: Conceptual changes in a living-donor program

Citation
M. Kasahara et al., Treatment of ornithine transcarbamylase deficiency in girls by auxiliary liver transplantation: Conceptual changes in a living-donor program, J PED SURG, 33(12), 1998, pp. 1753-1756
Citations number
16
Language
INGLESE
art.tipo
Article
Categorie Soggetti
Pediatrics
Journal title
JOURNAL OF PEDIATRIC SURGERY
ISSN journal
0022-3468 → ACNP
Volume
33
Issue
12
Year of publication
1998
Pages
1753 - 1756
Database
ISI
SICI code
0022-3468(199812)33:12<1753:TOOTDI>2.0.ZU;2-T
Abstract
Background/Purpose: Ornithine transcarbamylase (OTC) deficiency is an X-chr omosome-linked genetic disorder resulting in hyperammonemia hepatic dysfunc tion, coma, and serious neurological sequelae. This report describes an exp erience in treating this condition with living-related liver transplantatio n. Methods: Three children with OTC were treated with a living-related river t ransplantation. Potential donors were evaluated with the allopurinol loadin g test to exclude heterozygotes, FK506 and low-dose steroids were used for immunosuppression. Auxiliary partial orthotopic transplantation (APOLT) was used in two of the cases. Results: All three children survived and are doing well without protein res triction. Conclusions: These observations suggest that in OTC removal of the native l iver is not necessary because(1) a partial liver segment with normal enzyme activity corrects the hyperammonemia, (2) in case of graft failure the nat ive liver is an available backup, and (3) the native liver has the ability to recover function. As long as living-related donors serve as the main sou rce of donor organs, APOLT is a safer approach to liver transplantation in OTC. J Pediatr Surg 33:1753-1756 Copyright (C) 1998 by W.B. Saunders Compan y.