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Modified by the Innovative Drugs and Strategies-Pattern of Selected Indications for Pediatric Liver Transplantation
Irena Jankowska1, Piotr Socha1, Dorota Gliwicz1
1Department of Gastroenterology, Hepatology, Nutritional Disorders and Pediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
Insights
Pediatric liver transplantation (LTx) indications are evolving. New therapies are decreasing the need for LTx in some conditions while increasing it for others like urea cycle disorders.
Area of Science:
- Pediatric Hepatology
- Transplant Surgery
- Gastroenterology
Background:
- Liver transplantation (LTx) is a critical treatment for pediatric end-stage liver disease.
- Analysis of LTx registries reveals significant shifts in pediatric indication profiles.
Purpose of the Study:
- To review successes, hopes, and challenges of changing LTx indications in children.
- To analyze trends in pediatric liver transplantation based on literature and clinical experience.
Main Methods:
- Comprehensive literature review on pediatric LTx indications.
- Retrospective review of LTx indications at a tertiary pediatric hospital.
Main Results:
- Decreased LTx need for hepatitis B/C and tyrosinemia type 1 due to new therapies.
- RNAi therapy eliminated LTx for primary hyperoxaluria type 1.
- Increasing LTx for urea cycle disorders (UCDs) to prevent neurodevelopmental impairment.
Conclusions:
- Emerging therapies are altering the landscape of pediatric liver transplantation.
- Specific genetic and infectious liver diseases see reduced LTx requirements.
- Prophylactic LTx for UCDs is becoming more prevalent in pediatric care.
Background:
Liver transplantation (LTx) constitutes a major life-saving routine treatment for children with end-stage liver disease. However, the analysis of LTx registries in children provides much information about changes in the indication profiles in the recent years.
Methods:
The article provides a comprehensive review about the successes, hopes, and challenges related to changing indications for LTx in children based on the literature review and our own experience. Retrospective review of the indications for LTx at a tertiary referral pediatric hospital was also presented.
Results And Conclusions:
In the context of the new therapies that have emerged, the need for LTx has decreased in patients with chronic hepatitis B and C infection and tyrosinemia type 1. In primary hyperoxaluria type 1, new RNAi-based therapy has eliminated the requirement for LTx (both isolated or combined). There is a hope that introduction of ileal bile acid transporter (IBAT) blockers reduces the need for LTx in patients with Alagille syndrome or progressive familial intrahepatic cholestasis. The number of children qualified for LTx with urea cycle disorders (UCDs) as a prophylaxis of neurodevelopmental impairment is increasing.
