First European Case of Simultaneous Liver and Pancreas Transplantation as Treatment of Wolcott-Rallison Syndrome in a Small Child

Transplantation. 2020 Mar;104(3):522-525. doi: 10.1097/TP.0000000000002869.

Abstract

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.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child, Preschool
  • Diabetes Mellitus, Type 1 / complications
  • Diabetes Mellitus, Type 1 / diagnosis
  • Diabetes Mellitus, Type 1 / genetics
  • Diabetes Mellitus, Type 1 / surgery*
  • Epiphyses / abnormalities*
  • Epiphyses / surgery
  • Europe
  • Female
  • Genetic Testing
  • Humans
  • Liver Failure, Acute / etiology
  • Liver Failure, Acute / surgery*
  • Liver Transplantation / methods*
  • Osteochondrodysplasias / complications
  • Osteochondrodysplasias / diagnosis
  • Osteochondrodysplasias / genetics
  • Osteochondrodysplasias / surgery*
  • Pancreas Transplantation / methods*
  • Treatment Outcome
  • eIF-2 Kinase / genetics

Substances

  • EIF2AK3 protein, human
  • eIF-2 Kinase

Supplementary concepts

  • Wolcott-Rallison syndrome