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Pediatric Liver Disease Content  
Slide Library
   Released: 2022
Cholestatic Pruritus Across the Age Spectrum: What Are You Missing?
94 slides
 
 
   Released: 2021
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New Innovations for Children with Rare Cholestatic Liver Disease: Expert Perspectives on Diagnostic and Treatment Advances in PFIC and ALGS
61 slides
 
 
 
Webcast Library
     
Cholestatic Pruritus Across the Age Spectrum: What Are You Missing?   Educational Webcast
 
Clinical Advances in Wilson Disease: New Updates on Diagnosis, Management and Treatment.   Educational Webcast
 
Abstract Library
1.Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial
 

Lancet Gastroenterol Hepatol. 2022 Dec;7(12):1092-1102.doi: 10.1016/S2468-1253(22)00270-9. Epub 2022 Sep 30.

Michael L Schilsky 1, Anna Czlonkowska 2, Massimo Zuin 3, David Cassiman 4, Carlos Twardowschy 5, Aurelia Poujois 6, Francisco de Assis A Gondim 7, Gerald Denk 8, Rubens G Cury 9, Peter Ott 10, Joanna Moore 11, Aftab Ala 12, Renata D'Inca 13, Eduardo Couchonnal-Bedoya 14, Koenraad D'Hollander 15, Nicolas Dubois 15, C Omar F Kamlin 16, Karl Heinz Weiss 17; CHELATE trial investigators
 
2.Internal Ileal Diversion as Treatment for Progressive Familial Intrahepatic Cholestasis Type 1-Associated Graft Inflammation and Steatosis after Liver Transplantation
 

Children (Basel). 2022 Dec 14;9(12):1964. doi: 10.3390/children9121964.

Anna M Kavallar 1, Franka Messner 2, Stefan Scheidl 2, Rupert Oberhuber 2, Stefan Schneeberger 2, Denise Aldrian 1, Valeria Berchtold 2, Murat Sanal 2, Andreas Entenmann 1, Simon Straub 1, Anna Gasser 1, Andreas R Janecke 1 3, Thomas Müller 1, Georg F Vogel 1 4
 
3.Impact of liver dysfunction on outcomes in children with Alagille syndrome undergoing congenital heart surgery
 

Eur J Cardiothorac Surg. 2022 Dec 2;63(1):ezac553. doi: 10.1093/ejcts/ezac553.

Richard D Mainwaring 1, L Mac Felmly 1, R Thomas Collins 2, Frank L Hanley 1
 
4.Bilateral branch pulmonary arterial reconstruction in the Alagille syndrome: The technique and its pitfalls
 

Multimed Man Cardiothorac Surg. 2022 Dec 2;2022. doi: 10.1510/mmcts.2022.088.

Sameh M Said 1, Gamal Marey 2
 
5.Diagnosis and Outcomes of Late-Onset Wilson's Disease: A National Registry-Based Study
 

Mov Disord. 2022 Dec 27. doi: 10.1002/mds.29292. Online ahead of print.

Christelle Nilles 1 2, Mickael Alexandre Obadia 1 2, Rodolphe Sobesky 3, Jérôme Dumortier 4 5, Olivier Guillaud 4 6, Chloé Laurencin 4 7, Caroline Moreau 8, Claire Vanlemmens 9, Fabienne Ory-Magne 10, Victor de Ledinghen 11, Edouard Bardou-Jacquet 12, Frederique Fluchère 13, Corinne Collet 2 14, Nouzha Oussedik-Djebrani 2 15, France Woimant 2, Aurélia Poujois 1 2
 
6.Pseudocholinesterase as a Biomarker for Untreated Wilson's Disease
 

Biomolecules. 2022 Nov 30;12(12):1791. doi: 10.3390/biom12121791.

Harald Hefter 1, Max Arslan 1 2, Theodor S Kruschel 1, Max Novak 1, Dietmar Rosenthal 1, Sven G Meuth 1, Philipp Albrecht 1, Christian J Hartmann 1, Sara Samadzadeh 1 3
 
7.An Update on the Future of Wilson Disease Management
 

Prim Care Companion CNS Disord. 2022 Nov 29;24(6):AN21065COM1C.doi: 10.4088/PCC.AN21065COM1C.

Robert S Brown Jr 1
 
8.Sex differences and risk factors for bleeding in Alagille syndrome
 

EMBO Mol Med. 2022 Nov 8;e15809. doi: 10.15252/emmm.202215809. Online ahead of print.

Simona Hankeova 1 2, Noemi Van Hul 1, Jakub Laznovsky 3, Elisabeth Verboven 1, Katrin Mangold 1, Naomi Hensens 1 4, Csaba Adori 5, Elvira Verhoef 1 4, Tomas Zikmund 3, Feven Dawit 6, Michaela Kavkova 3, Jakub Salplachta 3, Marika Sjöqvist 1, Bengt R Johansson 7, Mohamed G Hassan 8 9, Linda Fredriksson 10, Karsten Baumgärtel 11, Vitezslav Bryja 2, Urban Lendahl 1, Andrew Jheon 8, Florian Alten 12, Kristina Teär Fahnehjelm 13 14, Björn Fischler 6, Jozef Kaiser 3, Emma R Andersson 1
 
9.The cholestatic infant: updates on diagnosis and genetics
 

Curr Opin Pediatr. 2022 Oct 1;34(5):491-495.doi: 10.1097/MOP.0000000000001156. Epub 2022 Aug 3.

Andrew Wehrman 1, Christine K Lee
 
10. Odevixibat: a promising new treatment for progressive familial intrahepatic cholestasis
 

Expert Opin Pharmacother 2022 Oct 24. doi: 10.1080/14656566.2022.2140040.Online ahead of print.

Sarah M Bedoyan 1, Olya T Lovell 2, Simon P Horslen 3, James E Squires 
 
11. Cholestatic Itch: Our Current Understanding of Pathophysiology and Treatments
 

Am J Clin Dermatol. 2022 Sep;23(5):647-659. doi: 10.1007/s40257-022-00710-2.Epub 2022 Jul 28.

Ashley Vander Does 1, Cynthia Levy 2, Gil Yosipovitch 3
 
12.Healthcare Resource Utilization by Patients with Alagille Syndrome
 

J Pediatr. 2022 Sep 27;S0022-3476(22)00855-1. doi: 10.1016/j.jpeds.2022.09.033.Online ahead of print.

Noelle H Ebel 1, Andrea Goldstein 2, Robin Howard 2, Douglas B Mogul 2, Jessica R Marden 3, Annika Anderson 3, Katherine Gaburo 3, Noam Kirson 3, Philip Rosenthal 4
 
13.High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease
 

Mol Ther Methods Clin Dev. 2022 Jun 9;26:98-106. doi:10.1016/j.omtm.2022.06.001.eCollection 2022 Sep 8.

Oihana Murillo 1, Maria Collantes 2 3, Cristina Gazquez 1, Daniel Moreno 1, Ruben Hernandez-Alcoceba 1, Miren Barberia 1, Margarita Ecay 3, Blanche Tamarit 4, Anne Douar 4, Veronica Ferrer 4, Jean Philippe Combal 4, Ivan Peñuelas 2 3, Bernard Bénichou 4, Gloria Gonzalez-Aseguinolaza 1 5
 
14.Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine
 

Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947.doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.

Karl Heinz Weiss 1, Carlot Kruse 2, Nina Manolaki 3, Massimo Zuin 4, Peter Ferenci 5, Daphne van Scheppingen 2, Larissa Wijnberg 2, Constance E de Koning 6, Anil Dhawan 7
 
15.Maralixibat Treatment Response in Alagille Syndrome Is Associated with Improved Health-Related Quality of Life
 

J Pediatr. 2022 Sep 9;S0022-3476(22)00791-0. doi: 10.1016/j.jpeds.2022.09.001.Online ahead of print.

Binita M Kamath 1, Andrea Goldstein 2, Robin Howard 2, Will Garner 2, Pamela Vig 2, Jessica R Marden 3, Emma Billmyer 3, Annika Anderson 3, Noam Kirson 3, Emmanuel Jacquemin 4, Emmanuel Gonzales 4
 
16.Validation of the PRUCISION Instruments in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis
 

Adv Ther. 2022 Sep 6. doi: 10.1007/s12325-022-02262-7. Online ahead of print.

Chad Gwaltney 1, Cristina Ivanescu 2, Lisa Karlsson 3, Natalie Warholic 3, Lise Kjems 3, Patrick Horn 3
 
17.Wilson Disease in Children
 

Clin Liver Dis. 2022 Aug;26(3):473-488. doi: 10.1016/j.cld.2022.03.008.Epub 2022 Jun 25.

Nanda Kerkar 1, Ajay Rana 2
 
18.Neuroimaging Correlates of Cognitive Deficits in Wilson's Disease
 

Mov Disord 2022 Aug;37(8):1728-1738. doi: 10.1002/mds.29123. Epub 2022 Jun 20.

Samuel Shribman 1, Maggie Burrows 1, Rhian Convery 2, Martina Bocchetta 2, Carole H Sudre 3 4 5, Julio Acosta-Cabronero 6, David L Thomas 2 7 8, Godfrey T Gillett 9, Emmanuel A Tsochatzis 10, Oliver Bandmann 11, Jonathan D Rohrer 2, Thomas T Warner 1
 
19.The Maternal and Fetal Outcomes of Pregnancy in Wilson's Disease: A Systematic Literature Review and Meta-Analysis
 

Biomedicines. 2022 Aug 24;10(9):2072. doi: 10.3390/biomedicines10092072.

Tomasz Litwin 1, Jan Bembenek 2, Agnieszka Antos 1, Iwona Kurkowska-Jastrzebska 1, Adam Przybylkowski 3, Marta Skowronska 1, Lukasz Smolinski 1, Anna Czlonkowska 1
 
20.Overview of Progressive Familial Intrahepatic Cholestasis
 

Clin Liver Dis. 2022 Aug;26(3):371-390. doi: 10.1016/j.cld.2022.03.003.

Sara Hassan 1, Paula Hertel 2
 
21.Development of the Patient- and Observer-Reported PRUCISION Instruments to Assess Pruritus and Sleep Disturbance in Pediatric Patients with Cholestatic Liver Diseases
 

Adv Ther. 2022 Sep 6. doi: 10.1007/s12325-022-02261-8. Online ahead of print.

Chad Gwaltney 1, Stephanie Bean 2, Meredith Venerus 3, Lisa Karlsson 4, Natalie Warholic 4, Lise Kjems 4, Patrick Horn 4
 
22.Natural History of Liver Disease in a Large International Cohort of Children with Alagille syndrome: Results from The GALA Study
 

Hepatology. 2022 Aug 29. doi: 10.1002/hep.32761. Online ahead of print.

Shannon M Vandriel 1, Li-Ting Li 2, Huiyu She 2, Jian-She Wang 2, Melissa A Gilbert 3, Irena Jankowska 4, Piotr Czubkowski 4, Dorota Gliwicz-Miedzinska 4, Emmanuel M Gonzales 5, Emmanuel Jacquemin 5, Jérôme Bouligand 6, Nancy B Spinner 3, Kathleen M Loomes, David A Piccoli, Lorenzo D'Antiga 7, Emanuele Nicastro 7, Étienne Sokal 8, Tanguy Demaret 8, Noelle H Ebel 9, Jeffrey A Feinstein 10, Rima Fawaz 11, Silvia Nastasio 12, Florence Lacaille 13, Dominique Debray 14, Henrik Arnell 15, Björn Fischler 16, Susan Siew 17, Michael Stormon 17, Saul J Karpen 18, Rene Romero 18, Kyung Mo Kim 19, Woo Yim Baek 19, Winita Hardikar 20, Sahana Shankar 21, Amin J Roberts 22, Helen M Evans 22, M Kyle Jensen 23, Marianne Kavan 23, Shikha S Sundaram 24, Alexander Chaidez 24, Palaniswamy Karthikeyan 25, Maria Camila Sanchez 26, Maria Lorena Cavalieri 26, Henkjan J Verkade 27, Way Seah Lee 28, James E Squires 29, Christina Hajinicolaou 30, Chatmanee Lertudomphonwanit 31, Ryan T Fischer 32, Catherine Larson-Nath 33, Yael Mozer-Glassberg 34, Cigdem Arikan 35, Henry C Lin 36, Jesus Quintero Bernabeu 37, Seema Alam 38, Deirdre Kelly 39, Elisa Carvalho 40, Cristina Targa Ferreira 41, Giuseppe Indolfi 42, Ruben E Quiros-Tejeira 43, Pinar Bulut 44, Pier Luigi Calvo 45, Zerrin Önal 46, Pamela L Valentino 11, Dev M Desai 47, John Eshun 48, Maria Rogalidou 49, Antal Dezsofi 50, Sabina Wiecek 51, Gabriella Nebbia 52, Raquel Borges Pinto 53, Victorien M Wolters 54, María Legarda Tamara 55, Andréanne N Zizzo 56, Jennifer Garcia 57<
 
23.Alagille Syndrome: Current Understanding of Pathogenesis, and Challenges in Diagnosis and Management
 

Clin Liver Dis. 2022 Aug;26(3):355-370. doi: 10.1016/j.cld.2022.03.002.Epub 2022 Jun 25.

Mohammed D Ayoub 1, Binita M Kamath 2
 
24.The cholestatic infant: updates on diagnosis and genetics
 

Curr Opin Pediatr. 2022 Oct 1;34(5):491-495.doi: 10.1097/MOP.0000000000001156. Epub 2022 Aug 3.

Andrew Wehrman 1, Christine K Lee
 
25.Epidemiology, treatment and burden of Wilson Disease in France: a 10-year analysis of the National Health Insurance Database
 

Clin Res Hepatol Gastroenterol. 2022 Jul 3;101992. doi: 10.1016/j.clinre.2022.101992.Online ahead of print.

Thomas Daniel-Robin 1, Bernard Bénichou 2, Claire Leboucher 3, Cécile Blein 3, Jean-Philippe Combal 2
 
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