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Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement

Background & Aims: Biliary atresia (BA) is the commonest single etiology indication for liver replacement in children. As timely access to liver transplantation (LT) remains challenging for small BA children (with prolonged waiting time being associated with clinical deterioration leading to bot...

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Autores principales: de Ville de Goyet, Jean, Illhardt, Toni, Chardot, Christophe, Dike, Peace N., Baumann, Ulrich, Brandt, Katherine, Wildhaber, Barbara E., Pakarinen, Mikko, di Francesco, Fabrizio, Sturm, Ekkehard, Cornet, Marianna, Lemoine, Caroline, Pfister, Eva Doreen, Calinescu, Ana M., Hukkinen, Maria, Harpavat, Sanjiv, Tuzzolino, Fabio, Superina, Riccardo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9032543/
https://www.ncbi.nlm.nih.gov/pubmed/35456234
http://dx.doi.org/10.3390/jcm11082142
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author de Ville de Goyet, Jean
Illhardt, Toni
Chardot, Christophe
Dike, Peace N.
Baumann, Ulrich
Brandt, Katherine
Wildhaber, Barbara E.
Pakarinen, Mikko
di Francesco, Fabrizio
Sturm, Ekkehard
Cornet, Marianna
Lemoine, Caroline
Pfister, Eva Doreen
Calinescu, Ana M.
Hukkinen, Maria
Harpavat, Sanjiv
Tuzzolino, Fabio
Superina, Riccardo
author_facet de Ville de Goyet, Jean
Illhardt, Toni
Chardot, Christophe
Dike, Peace N.
Baumann, Ulrich
Brandt, Katherine
Wildhaber, Barbara E.
Pakarinen, Mikko
di Francesco, Fabrizio
Sturm, Ekkehard
Cornet, Marianna
Lemoine, Caroline
Pfister, Eva Doreen
Calinescu, Ana M.
Hukkinen, Maria
Harpavat, Sanjiv
Tuzzolino, Fabio
Superina, Riccardo
author_sort de Ville de Goyet, Jean
collection PubMed
description Background & Aims: Biliary atresia (BA) is the commonest single etiology indication for liver replacement in children. As timely access to liver transplantation (LT) remains challenging for small BA children (with prolonged waiting time being associated with clinical deterioration leading to both preventable pre- and post-transplant morbidity and mortality), the care pathway of BA children in need of LT was analyzed—from diagnosis to LT—with particular attention to referral patterns, timing of referral, waiting list dynamics and need for medical assistance before LT. Methods: International multicentric retrospective study. Intent-to-transplant study analyzing BA children who had indication for LT early in life (aged < 3 years at the time of assessment), over the last 5 years (2016–2020). Clinical and laboratory data of 219 BA children were collected from 8 transplant centers (6 in Europe and 2 in USA). Results: 39 patients underwent primary transplants. Children who underwent Kasai in a specialist -but not transplant- center were older at time of referral and at transplant. At assessment for LT, the vast majority of children already were experiencing complication of cirrhosis, and the majority of children needed medical assistance (nutritional support, hospitalization, transfusion of albumin or blood) while waiting for transplantation. Severe worsening of the clinical condition led to the need for requesting a priority status (i.e., Peld Score exception or similar) for timely graft allocation for 76 children, overall (35%). Conclusions: As LT currently results in BA patient survival exceeding 95% in many expert LT centers, the paradigm for BA management optimization and survival have currently shifted to the pre-LT management. The creation of networks dedicated to the timely referral to a pediatric transplant center and possibly centralization of care should be considered, in combination with implementing all different graft type surgeries in specialist centers (including split and living donor LTs) to achieve timely LT in this vulnerable population.
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spelling pubmed-90325432022-04-23 Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement de Ville de Goyet, Jean Illhardt, Toni Chardot, Christophe Dike, Peace N. Baumann, Ulrich Brandt, Katherine Wildhaber, Barbara E. Pakarinen, Mikko di Francesco, Fabrizio Sturm, Ekkehard Cornet, Marianna Lemoine, Caroline Pfister, Eva Doreen Calinescu, Ana M. Hukkinen, Maria Harpavat, Sanjiv Tuzzolino, Fabio Superina, Riccardo J Clin Med Article Background & Aims: Biliary atresia (BA) is the commonest single etiology indication for liver replacement in children. As timely access to liver transplantation (LT) remains challenging for small BA children (with prolonged waiting time being associated with clinical deterioration leading to both preventable pre- and post-transplant morbidity and mortality), the care pathway of BA children in need of LT was analyzed—from diagnosis to LT—with particular attention to referral patterns, timing of referral, waiting list dynamics and need for medical assistance before LT. Methods: International multicentric retrospective study. Intent-to-transplant study analyzing BA children who had indication for LT early in life (aged < 3 years at the time of assessment), over the last 5 years (2016–2020). Clinical and laboratory data of 219 BA children were collected from 8 transplant centers (6 in Europe and 2 in USA). Results: 39 patients underwent primary transplants. Children who underwent Kasai in a specialist -but not transplant- center were older at time of referral and at transplant. At assessment for LT, the vast majority of children already were experiencing complication of cirrhosis, and the majority of children needed medical assistance (nutritional support, hospitalization, transfusion of albumin or blood) while waiting for transplantation. Severe worsening of the clinical condition led to the need for requesting a priority status (i.e., Peld Score exception or similar) for timely graft allocation for 76 children, overall (35%). Conclusions: As LT currently results in BA patient survival exceeding 95% in many expert LT centers, the paradigm for BA management optimization and survival have currently shifted to the pre-LT management. The creation of networks dedicated to the timely referral to a pediatric transplant center and possibly centralization of care should be considered, in combination with implementing all different graft type surgeries in specialist centers (including split and living donor LTs) to achieve timely LT in this vulnerable population. MDPI 2022-04-12 /pmc/articles/PMC9032543/ /pubmed/35456234 http://dx.doi.org/10.3390/jcm11082142 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
de Ville de Goyet, Jean
Illhardt, Toni
Chardot, Christophe
Dike, Peace N.
Baumann, Ulrich
Brandt, Katherine
Wildhaber, Barbara E.
Pakarinen, Mikko
di Francesco, Fabrizio
Sturm, Ekkehard
Cornet, Marianna
Lemoine, Caroline
Pfister, Eva Doreen
Calinescu, Ana M.
Hukkinen, Maria
Harpavat, Sanjiv
Tuzzolino, Fabio
Superina, Riccardo
Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title_full Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title_fullStr Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title_full_unstemmed Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title_short Variability of Care and Access to Transplantation for Children with Biliary Atresia Who Need a Liver Replacement
title_sort variability of care and access to transplantation for children with biliary atresia who need a liver replacement
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9032543/
https://www.ncbi.nlm.nih.gov/pubmed/35456234
http://dx.doi.org/10.3390/jcm11082142
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