Managing recurrent portal steal in auxiliary liver transplantation for non-cirrhotic metabolic liver disease.
CTLN1
NCMLD
PS
PVE
gene therapy
metabolic profile
Journal
Pediatric transplantation
ISSN: 1399-3046
Titre abrégé: Pediatr Transplant
Pays: Denmark
ID NLM: 9802574
Informations de publication
Date de publication:
12 2022
12 2022
Historique:
revised:
01
07
2022
received:
18
05
2022
accepted:
23
08
2022
pubmed:
15
9
2022
medline:
18
11
2022
entrez:
14
9
2022
Statut:
ppublish
Résumé
APOLT has been proposed as a treatment modality for certain types of NCMLD. While the short-term outcomes of this operation have been comparable with orthotopic LT, its long-term outcomes have sparsely been reported. We present one such case of Citrullinemia type I who underwent APOLT and developed recurrent PS. A 2-year-old male child with a diagnosis of Citrullinemia type I underwent APOLT with a left lateral segment from a split deceased donor liver, and his postoperative period was unremarkable. Ammonia-lowering agents were stopped 1 week following the operation and the child was discharged home on a normal diet. Four years following APOLT, the child presented with altered sensorium and seizures. A diagnosis of PS was made. Subsequent to an embolization of the native liver's right anterior portal vein his sensorium improved and he remained clinically stable on a normal diet. Six years following the APOLT, the child again presented with features of acute encephalopathy. Imaging was suggestive of PS. A portal vein embolization of the native portal vein was performed and the child's clinical condition improved. At 6 months' follow-up, the child remains well on a normal diet. While the early impediments in this technique may have been overcome, in the absence of any realistic clinical application gene therapy, the debate of long-term phenotypic metabolic correction for NCMLD by APOLT needs to be revisited.
Sections du résumé
BACKGROUND
APOLT has been proposed as a treatment modality for certain types of NCMLD. While the short-term outcomes of this operation have been comparable with orthotopic LT, its long-term outcomes have sparsely been reported. We present one such case of Citrullinemia type I who underwent APOLT and developed recurrent PS.
CASE REPORT
A 2-year-old male child with a diagnosis of Citrullinemia type I underwent APOLT with a left lateral segment from a split deceased donor liver, and his postoperative period was unremarkable. Ammonia-lowering agents were stopped 1 week following the operation and the child was discharged home on a normal diet. Four years following APOLT, the child presented with altered sensorium and seizures. A diagnosis of PS was made. Subsequent to an embolization of the native liver's right anterior portal vein his sensorium improved and he remained clinically stable on a normal diet. Six years following the APOLT, the child again presented with features of acute encephalopathy. Imaging was suggestive of PS. A portal vein embolization of the native portal vein was performed and the child's clinical condition improved. At 6 months' follow-up, the child remains well on a normal diet.
CONCLUSIONS
While the early impediments in this technique may have been overcome, in the absence of any realistic clinical application gene therapy, the debate of long-term phenotypic metabolic correction for NCMLD by APOLT needs to be revisited.
Types de publication
Case Reports
Langues
eng
Sous-ensembles de citation
IM
Pagination
e14389Informations de copyright
© 2022 Wiley Periodicals LLC.
Références
Vara R, Dhawan A, Deheragoda M, et al. Liver transplantation for neonatal onset citrullinemia. Pediatr Transplant. 2018;22(2):e13191.
Rammohan A, Reddy MS, Narasimhan G, et al. Auxillary partial orthotopic liver transplantation for selected non cirrhotic metabolic liver disease: no longer stepping through the looking glass? Liver Transpl. 2018;25:111-118.
Shankar S, Valamparampil J, Rammohan A, et al. Minimally invasive treatment of metabolic decompensation due to portal steal in auxillary liver transplantation. Liver Transpl. 2019;25:960-963.
Morioka D, Kasahara M, Takada Y, et al. Current role of liver transplantation for the treatment of urea cycle disorders: a review of the worldwide English literature and 13 cases at Kyoto University. Liver Transpl. 2005;11:1332-1342.
Reddy MS, Rajalingam R, Rela M. Revisiting APOLT for metabolic liver disease: a new look at an old idea. Transplantation. 2017;101:260-266.
Lee B, Dennis JA, Healy JP, et al. Hepatocyte gene therapy in a large animal: a neonatal bovine model of citrullinemia. Proc Natl Acad Sci USA. 1999;96:3981-3986.