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Pediatric liver transplantation for inherited metabolic disease-Current challenges

Roshni Vara1,2, Nedim Hadzic2

  • 1Department of Metabolic Medicine, Evelina Children's Hospital, London, UK.

Saudi Journal of Gastroenterology : Official Journal of the Saudi Gastroenterology Association
|March 7, 2026
PubMed

Insights

Liver transplantation (LT) has evolved significantly, offering over 90% survival for pediatric patients. It is increasingly vital for treating inherited metabolic diseases (IMDs), providing curative or corrective options.

Area of Science:

  • Hepatology and Transplant Surgery
  • Pediatric Gastroenterology and Hepatology
  • Medical Genetics and Metabolism

Background:

  • Pediatric liver transplantation (LT) faced early challenges but saw significant improvements in surgical techniques and immunosuppression from the 1970s onwards.
  • Current 10-year survival rates for elective pediatric LT exceed 90%, demonstrating substantial progress in the field.
  • Inherited metabolic diseases (IMDs) now represent a major indication for pediatric LT, accounting for 25-30% of cases.

Purpose of the Study:

  • To review the evolving role of liver transplantation in managing inherited metabolic diseases in children.
  • To highlight current concepts, challenges, and controversies surrounding LT for IMDs.
  • To examine the curative and corrective potential of LT in various IMDs.

Main Methods:

  • Review of historical data and advancements in pediatric LT techniques and immunosuppressive therapies.
  • Analysis of the increasing utilization of LT for IMDs since the late 1970s.
  • Examination of specific IMDs where LT is indicated, including tyrosinaemia type 1 and urea cycle disorders.

Main Results:

  • Pediatric LT outcomes have dramatically improved, with high long-term graft and patient survival rates.
  • LT is established as a curative treatment for IMDs with liver-confined defects and partially corrective for those with extrahepatic involvement.
  • The scope of LT for IMDs has expanded, with earlier interventions being emphasized.

Conclusions:

  • Liver transplantation is a critical therapeutic option for pediatric patients with inherited metabolic diseases.
  • Continued advancements are enhancing the efficacy and safety of LT for IMDs.
  • The expanding indications and emphasis on early intervention necessitate ongoing research into challenges and controversies in pediatric LT for IMDs.
Abstract

No abstract available in PubMed .

Keywords:
Inherited metabolic diseaseliver transplantationpediatric

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