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Review
. 2024 Aug;28(5):e14825.
doi: 10.1111/petr.14825.

Modified by the Innovative Drugs and Strategies-Pattern of Selected Indications for Pediatric Liver Transplantation

Irena Jankowska  1 , Piotr Socha  1 , Dorota Gliwicz  1 , Patryk Lipiński  2   3 , Dariusz Rokicki  2 , Piotr Kaliciński  4 , Ewa Danielewska  4 , Ryszard Grenda  5
Affiliations
Review

Modified by the Innovative Drugs and Strategies-Pattern of Selected Indications for Pediatric Liver Transplantation

Irena Jankowska et al. Pediatr Transplant. 2024 Aug.

Abstract

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.

Keywords: children; ileal bile acid transporter (IBAT) blockers; liver metabolic diseases; liver transplantation; primary hyperoxaluria type 1.

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References

    1. U. Baumann, V. Karam, R. Adam, et al., “European Liver and Intestine Transplant Association (ELITA) and All ELTR Contributing Centers. Prognosis of Children Undergoing Liver Transplantation: A 30‐Year European Study,” Pediatrics 150 (2022): e2022057424.
    1. D. Haldar, B. Kern, J. Hodson, et al., “European Liver and Intestine Transplant Association (ELITA). Outcomes of Liver Transplantation for Non‐Alcoholic Steatohepatitis: A European Liver Transplant Registry Study,” Journal of Hepatology 71 (2019): 313–322.
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    1. R. Belostotsky and Y. Frishberg, “Novel Therapeutic Approaches for the Primary Hyperoxalurias,” Pediatric Nephrology 36 (2021): 2593–2606.
    1. T. Cargill and E. Barnes, “Therapeutic Vaccination for Treatment of Chronic Hepatitis B,” Clinical and Experimental Immunology 205 (2021): 106–118.

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