First European Case of Simultaneous Liver and Pancreas Transplantation as Treatment of Wolcott-Rallison Syndrome in a Small Child.
Child, Preschool
Diabetes Mellitus, Type 1
/ complications
Epiphyses
/ abnormalities
Europe
Female
Genetic Testing
Humans
Liver Failure, Acute
/ etiology
Liver Transplantation
/ methods
Osteochondrodysplasias
/ complications
Pancreas Transplantation
/ methods
Treatment Outcome
eIF-2 Kinase
/ genetics
Journal
Transplantation
ISSN: 1534-6080
Titre abrégé: Transplantation
Pays: United States
ID NLM: 0132144
Informations de publication
Date de publication:
03 2020
03 2020
Historique:
pubmed:
25
7
2019
medline:
2
10
2020
entrez:
24
7
2019
Statut:
ppublish
Résumé
The concept of organ transplantation as treatment for complex genetic conditions, including Wolcott-Rallison syndrome (WRS), continues to show promise. Liver transplantation is essential for survival of patients with WRS, and pancreas transplantation cures their type I diabetes mellitus. The recipient, a 3-year-old girl weighing 14 kg at the time of transplantation, suffered from major complications of WRS, including repetitive liver failure episodes and poorly controlled diabetes. The patient underwent a nonacute, combined, simultaneous liver and pancreas transplantation from a pediatric donor without using the en bloc technique. Well-preserved graft functions at 2-year follow-up with normal liver and pancreas function. This is the first case report of simultaneous liver and pancreas transplantation as treatment of WRS in a small child in Europe. Two-year follow-up demonstrates that organ transplantation can halt life-threating recurrent liver failure episodes and cure type 1 diabetes.
Sections du résumé
BACKGROUND
The concept of organ transplantation as treatment for complex genetic conditions, including Wolcott-Rallison syndrome (WRS), continues to show promise. Liver transplantation is essential for survival of patients with WRS, and pancreas transplantation cures their type I diabetes mellitus.
METHODS
The recipient, a 3-year-old girl weighing 14 kg at the time of transplantation, suffered from major complications of WRS, including repetitive liver failure episodes and poorly controlled diabetes. The patient underwent a nonacute, combined, simultaneous liver and pancreas transplantation from a pediatric donor without using the en bloc technique.
RESULTS
Well-preserved graft functions at 2-year follow-up with normal liver and pancreas function.
CONCLUSIONS
This is the first case report of simultaneous liver and pancreas transplantation as treatment of WRS in a small child in Europe. Two-year follow-up demonstrates that organ transplantation can halt life-threating recurrent liver failure episodes and cure type 1 diabetes.
Identifiants
pubmed: 31335762
doi: 10.1097/TP.0000000000002869
pii: 00007890-202003000-00018
doi:
Substances chimiques
EIF2AK3 protein, human
EC 2.7.11.1
eIF-2 Kinase
EC 2.7.11.1
Types de publication
Case Reports
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
522-525Références
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