Review
Gastroenterology & Hepatology
Rishi Bolia, Akhil Dhanesh Goel, Vishakha Sharma, Anshu Srivastava
Summary: Biliary diversion is effective in relieving pruritus in PFIC, with PIBD having lower complications and LT requirements compared to PEBD.
EXPERT REVIEW OF GASTROENTEROLOGY & HEPATOLOGY
(2022)
Review
Pediatrics
Maria Noelle Huepper, Judith Pichler, Wolf-Dietrich Huber, Andreas Heilos, Rebecca Schaup, Martin Metzelder, Sophie Langer
Summary: This study surveyed and reviewed the etiology and treatments for progressive familial intrahepatic cholestasis (PFIC), finding that surgical biliary diversion and ileal bile salt inhibitors can delay or prevent liver transplantation, and a combination of the two may be possible. Further research is warranted.
Review
Medicine, General & Internal
Hafiza Sidra Tul Muntaha, Mubashar Munir, Syeda Haleema Sajid, Zouina Sarfraz, Azza Sarfraz, Karla Robles-Velasco, Muzna Sarfraz, Miguel Felix, Ivan Cherrez-Ojeda
Summary: This study provides the latest evidence for the efficacy of ileal bile acid transport (IBAT) blockers in patients with Alagille syndrome (ALGS). The results demonstrate that these drugs can significantly reduce itch and serum bile acid levels, while improving fatigue and quality of life. However, these drugs may lead to elevated levels of alanine aminotransferase (ALT).
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Gastroenterology & Hepatology
Ronald J. Sokol, Emmanuel M. Gonzales, Binita M. Kamath, Alastair Baker, Pamela Vig, Douglas B. Mogul, Will Garner, Bettina E. Hansen, Emmanuel Jacquemin, Richard J. Thompson
Summary: Improvement in pruritus by 48 weeks, and lower W48 bilirubin and serum bile acid levels were associated with fewer events in patients with Alagille syndrome (ALGS) treated with maralixibat (MRX).
Article
Gastroenterology & Hepatology
Angelo Di Giorgio, Marco Sciveres, Maurizio Fuoti, Aurelio Sonzogni, Claudia Mandato, Lorenzo D'Antiga
Summary: This case series describes the use of odevixibat, a selective IBAT inhibitor, in children with TJP2 deficiency. After treatment, there was a significant decrease in serum bile acids levels and improvement in pruritus. Odevixibat was well tolerated and can be considered as a valuable treatment option for patients with TJP2 deficiency.
CLINICS AND RESEARCH IN HEPATOLOGY AND GASTROENTEROLOGY
(2023)
Review
Medicine, General & Internal
Jacqueline A. G. M. Langedijk, Ulrich H. Beuers, Ronald P. J. Oude Elferink
Summary: This article discusses the debilitating symptom of pruritus in various cholestatic disorders, the unresolved molecular mechanisms, and the current guidelines-approved therapy. Experimental therapeutic approaches are also necessary to improve patients' quality of life.
FRONTIERS IN MEDICINE
(2021)
Review
Biochemistry & Molecular Biology
Jin-Cheol Kim, Won-Sik Shim, In-Suk Kwak, Dong-Hun Lee, Jin-Seo Park, So-Yeon Lee, Seok-Young Kang, Bo-Young Chung, Chun-Wook Park, Hye-One Kim
Summary: This article summarizes the causes, pathophysiology, and appropriate treatments of pruritus caused by systemic diseases, with a focus on chronic kidney disease and cholestatic liver disease. It contributes to improving the quality of life for patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Pediatrics
Kristin Bjornland, Maria Hukkinen, Vladimir Gatzinsky, Henrik Arnell, Mikko P. Pakarinen, Runar Almaas, Jan F. Svensson
Summary: In children with rare cholestatic liver diseases, partial biliary diversion (PBD) may provide long-term relief from debilitating pruritus and preserve native liver function, although stoma-related complications and reoperations are common.
EUROPEAN JOURNAL OF PEDIATRIC SURGERY
(2021)
Article
Surgery
Abdulla Sahloul, Elke Lainka, Simone Kathemann, Sandra Swoboda, Carola Droege, Verena Keitel, Yahya Saleh Al-Matary, Michael Berger, Maren Schulze
Summary: In our retrospective analysis, we found that biliary diversion effectively reduced bile acids, cholesterol levels, and triglycerides in children with PFIC 1, but not in those with PFIC 2. Furthermore, a decrease in bile acids following biliary diversion predicted the need for liver transplantation.
FRONTIERS IN SURGERY
(2023)
Review
Gastroenterology & Hepatology
Huasheng Yu, Kirk Wangensteen, Tong Deng, Yulong Li, Wenqin Luo
Summary: This passage discusses the debilitating symptom of pruritus in liver diseases with cholestasis and the recent identification of MRGPRX4 as a receptor for bile acids and bilirubin in cholestatic itch. It summarizes current theories and knowledge of cholestatic itch, emphasizing MRGPRX4 as a key mediator and discussing future perspectives in this area of research.
SEMINARS IN LIVER DISEASE
(2021)
Review
Gastroenterology & Hepatology
Rebecca Roediger, Jaquelyn Fleckenstein
Summary: Intrahepatic cholestasis of pregnancy (ICP) is a common disorder in the second half of pregnancy with risks of adverse effects on the fetus. Although genetically susceptible, there is currently no proven preventive method, and ursodeoxycholic acid remains the first-line treatment option.
SEMINARS IN LIVER DISEASE
(2021)
Review
Biology
Minna Rodrigo, Xinzhong Dong, Daphne Chien, Wikrom Karnsakul
Summary: Pediatric patients with liver disease commonly experience itching, which can significantly affect their quality of life. The cause of itching in liver disease is not fully known and may be caused by many factors. Treating this symptom can be difficult and may require surgical interventions in certain cases. Understanding the cause of itching may help identify better treatment options.
Article
Medicine, General & Internal
Emmanuel Gonzales, Winita Hardikar, Michael Stormon, Alastair Baker, Loreto Hierro, Dorota Gliwicz, Florence Lacaille, Alain Lachaux, Ekkehard Sturm, Kenneth D. R. Setchell, Ciara Kennedy, Alejandro Dorenbaum, Jana Steinmetz, Nirav K. Desai, Andrew J. Wardle, Will Garner, Pamela Vig, Thomas Jaecklin, Etienne M. Sokal, Emmanuel Jacquemin
Summary: Maralixibat showed safety and efficacy in children with Alagille syndrome, providing durable and clinically meaningful improvements in cholestasis. It may represent a new treatment paradigm for chronic cholestasis in Alagille syndrome.
Article
Gastroenterology & Hepatology
Ranya Selim, Joseph Ahn
Summary: Pruritus associated with chronic liver disease has a complex and not fully understood pathophysiology. For patients whose quality of life is affected by severe itching, liver transplantation may be considered if medical therapy fails to provide relief. Further research is needed to fully understand the pathophysiology of pruritus in chronic liver disease, which would help in developing more effective treatments for this condition.
CLINICS IN LIVER DISEASE
(2023)
Article
Gastroenterology & Hepatology
Cynthia Levy, Michael Manns, Gideon Hirschfield
Summary: Primary biliary cholangitis is an autoimmune disease with symptoms including fatigue, itch, abdominal pain, and sicca complex. Treatment focuses on cholestatic consequences.
CLINICAL GASTROENTEROLOGY AND HEPATOLOGY
(2023)
Article
Pediatrics
Alastair Baker, Esteban Frauca Remacha, Juan Torres Canizales, Luz Yadira Bravo-Gallego, Emer Fitzpatrick, Angel Alonso Melgar, Gema Munoz Bartolo, Luis Garcia Guereta, Esther Ramos Boluda, Yasmina Mozo, Dorota Broniszczak, Wioletta Jarmuzek, Piotr Kalicinski, Britta Maecker-Kolhoff, Julia Carlens, Ulrich Baumann, Charlotte Roy, Christophe Chardot, Elisa Benetti, Mara Cananzi, Elisabetta Calore, Luca Dello Strologo, Manila Candusso, Maria Francelina Lopes, Manuel Joao Brito, Cristina Goncalves, Carmen Do Carmo, Xavier Stephenne, Lars Wennberg, Rosario Stone, Jelena Rascon, Caroline Lindemans, Dominik Turkiewicz, Eugenia Giraldi, Emanuele Nicastro, Lorenzo D'Antiga, Oanez Ackermann, Paloma Jara Vega
Summary: A cross-sectional questionnaire study was conducted in 13 European centers with 34 SOT programs on PTLD practices, revealing that decisions for preemptive treatment were often based on EBV viremia monitoring and clinical assessment, while reducing immunosuppression was a common initial treatment modality at diagnosis. A total of 126 PTLD cases were reported from 2012 to 2016, with an 88% survival rate among diagnosed cases.
Article
Pediatrics
Malgorzata Markiewicz-Kijewska, Piotr Kalicinski, Juan Torres Canizales, Angelo Di Giorgio, Ulrich Baumann, Carl Jorns, Alastair Baker, Maria Francelina Lopes, Esteban Frauca Remacha, Eduardo Lopez-Granados, Paloma Jara Vega, Maria-Sole Basso, Grzegorz Kowalewski, Diana Kaminska, Sandra Ferreira, Daniela Liccardo, Andrea Pietrobattista, Marco Spada
Summary: AB0-incompatible (AB0i) liver transplantations in pediatric patients have been found to be affected significantly by donor type, urgency of transplantation, and development of vascular complications. A longer time from the first AB0i LT in the study results in better outcomes, indicating an international learning experience.
Article
Pediatrics
Malgorzata Markiewicz-Kijewska, Sylwia Szymanska, Michal Pyzlak, Piotr Kalicinski, Joanna Teisseyre, Adam Kowalski, Irena Jankowska, Piotr Czubkowski, Hor Ismail
Summary: Liver transplantation has become a routine treatment for children with end stage liver failure, with long term survival improving. However, many patients showed pathomorphological abnormalities in late protocol biopsies despite normal laboratory liver tests, indicating a slow subclinical process. The study supports the need for protocol liver biopsies even in patients with normal/almost normal biochemical liver tests.
Article
Pediatrics
Marek Stefanowicz, Maria Janowska, Joanna Pawlowska, Anna Tylki-Szymanska, Adam Kowalski, Marek Szymczak, Piotr Kalicinski, Irena Jankowska
Summary: Transaldolase deficiency is a rare genetic disorder that causes liver and kidney damage. Liver transplantation is a treatment option that can be effective in improving quality of life and long-term outcomes for patients with this condition.
Article
Pediatrics
Marek Stefanowicz, Grzegorz Kowalewski, Marek Szymczak, Waldemar Patkowski, Krzysztof Zieniewicz, Ireneusz Grzelak, Adam Kowalski, Hor Ismail, Diana Kaminska, Piotr Kalicinski
Summary: This study aimed to assess the impact of the graft-recipient weight ratio (GRWR) on early post-transplant complications and patient survival rates in children after living donor liver transplantation. It was found that recipients with a GRWR < 5% had significantly better prognosis concerning patients and graft survival. Delayed abdominal wound closure was more common in patients with a GRWR > 3.5%.
Article
Pediatrics
Grzegorz Kowalewski, Piotr Kalicinski, Marek Stefanowicz, Ryszard Grenda, Piotr Czubkowski, Marek Szymczak
Summary: Chronic kidney disease (CKD) is a common complication after liver transplantation (LT), especially in children, with varying prevalence based on transplantation era and immunosuppression regimens. Maintaining tacrolimus concentrations >4 ng/mL in long-term observation is associated with a significant increase in microalbuminuria. Microalbuminuria, a risk factor of CKD, emphasizes the necessity of regular comprehensive assessment for patients in long-term follow-up.
Article
Gastroenterology & Hepatology
Jean de Ville de Goyet, Ulrich Baumann, Vincent Karam, Rene Adam, Silvio Nadalin, Nigel Heaton, Raymond Reding, Sophie Branchereau, Darius Mirza, Juergen L. Klempnauer, Lutz Fischer, Piotr Kalicinski, Michele Colledan, Manuel Lopez Santamaria, Ruben H. de Kleine, Christophe Chardot, Sezai Yilmaz, Murat Kilic, Olivier Boillot, Fabrizio di Francesco, Wojciech G. Polak, Henkjan J. Verkade, European Liver
Summary: Through retrospective data analysis, it was found that the 5-year graft survival rate of liver transplants in European children has steadily increased, but the late annual graft loss rate in teenagers is relatively higher. Liver transplantation before or after puberty may impact the long-term outcome in children.
Article
Pediatrics
Tomasz Jarmolinski, Monika Rosa, Blanka Rybka, Renata Ryczan-Krawczyk, Kornelia Gajek, Katarzyna Bogunia-Kubik, Maja Klaudel-Dreszler, Piotr Czubkowski, Piotr Kalicinski, Joanna Teisseyre, Marek Stefanowicz, Ewa Gorczynska, Krzysztof Kalwak, Marek Ussowicz
Summary: A child with Fanconi anemia underwent orthotopic liver transplantation (OLT) after hematopoietic stem cell transplantation (HSCT) complicated by acute graft versus host disease (GVHD). The liver can serve as a clinically significant source of hematopoietic stem cells. Close monitoring of hematopoietic chimerism after OLT is necessary in patients at risk for complications such as GVHD.
FRONTIERS IN PEDIATRICS
(2022)
Article
Pediatrics
Marek Stefanowicz, Piotr Kalicinski, Grzegorz Kowalewski, Adam Kowalski, Mateusz Ciopinski, Marek Szymczak, Agnieszka Kwiecinska, Waldemar Patkowski, Krzysztof Zieniewicz, Ireneusz Grzelak, Diana Kaminska, Hor Ismail
Summary: The aim of this study was to analyze the risk factors for hepatic artery thrombosis (HAT) and its impact on long-term outcomes in pediatric living donor liver transplantation (LDLT). The study included 400 patients who underwent primary LDLT between 1999 and 2020. The results showed that acute liver failure, a hepatic artery anastomosis diameter below 2 mm, and intraoperative hepatic artery flow dysfunction were associated with a higher risk of HAT. Patients in the HAT group had worse survival rates and higher rates of biliary stenosis and retransplantation compared to the non-HAT group. Close monitoring of hepatic artery flow and immediate surgical revascularization can help mitigate the risks associated with HAT.
Article
Pediatrics
Adam Kowalski, Grzegorz Kowalewski, Piotr Kalicinski, Katarzyna Pankowska-Wozniak, Marek Szymczak, Hor Ismail, Marek Stefanowicz
Summary: Choledochal cyst is a rare malformation primarily diagnosed in children, and surgical resection followed by Roux-en-Y hepaticojejunostomy remains the most effective therapy. Treating asymptomatic neonates is still a topic of debate.
Article
Gastroenterology & Hepatology
Richard J. Thompson, Reha Artan, Ulrich Baumann, Pier Luigi Calvo, Piotr Czubkowski, Buket Dalgic, Lorenzo D'Antiga, Angelo Di Giorgio, Ozlem Durmaz, Emmanuel Gonzales, Tassos Grammatikopoulos, Girish Gupte, Winita Hardikar, Roderick H. J. Houwen, Binita M. Kamath, Saul J. Karpen, Florence Lacaille, Alain Lachaux, Elke Lainka, Kathleen M. Loomes, Cara L. Mack, Jan P. Mattsson, Patrick Mckiernan, Quanhong Ni, Hasan Ozen, Sanjay R. Rajwal, Bertrand Roquelaure, Eyal Shteyer, Etienne Sokal, Ronald J. Sokol, Nisreen Soufi, Ekkehard Sturm, Mary Elizabeth Tessier, Wendy L. van der Woerd, Henkjan J. Verkade, Jennifer M. Vittorio, Terese Wallefors, Natalie Warholic, Qifeng Yu, Patrick Horn, Lise Kjems
Summary: The ongoing PEDFIC 2 study evaluated the effectiveness of odevixibat in patients with progressive familial intrahepatic cholestasis. The study found that odevixibat can reduce serum bile acids and alleviate pruritus symptoms. This suggests that odevixibat may be a safe and effective treatment option for patients with progressive familial intrahepatic cholestasis.
Article
Pediatrics
Piotr Kalicinski, Ryszard Grenda, Marek Szymczak, Elzbieta Pietraszek, Joanna Pawlowska
Summary: This report describes the clinical pattern, diagnostic and therapeutic modalities in children with acute liver failure at risk of death without liver transplantation. The study emphasizes the difficulties in treating these children and highlights the importance of timely qualification and transplantation for their survival.
PEDIATRIC TRANSPLANTATION
(2023)
Meeting Abstract
Immunology
Dorota Broniszczak-Czyszek, Hor Ismail, Katarzyna Wozniak-Pankowska, Marek Szymczak, Jacek Rubik, Ryszard Grenda, Piotr Gastol, Malgorzata Syczewska, Piotr Kalicinski
Article
Genetics & Heredity
Jan Gehlen, Ann-Sophie Giel, Ricarda Koellges, Stephan L. Haas, Rong Zhang, Jiri Trcka, Ayse O. Sungur, Florian Renziehausen, Dorothea Bornholdt, Daphne Jung, Paul D. Hoyer, Agneta Nordenskjold, Dick Tibboel, John Vlot, Manon C. W. Spaander, Robert Smigiel, Dariusz Patkowski, Nel Roeleveld, Iris Alm van Rooij, Ivo de Blaauw, Alice Hoelscher, Marcus Pauly, Andreas Leutner, Joerg Fuchs, Joel Niethammer, Maria-Theodora Melissari, Ekkehart Jenetzky, Nadine Zwink, Holger Thiele, Alina Christine Hilger, Timo Hess, Jessica Trautmann, Matthias Marks, Martin Baumgarten, Gaby Blaess, Mikael Landen, Bengt Fundin, Cynthia M. Bulik, Tracie Pennimpede, Michael Ludwig, Kerstin U. Ludwig, Elisabeth Mangold, Stefanie Heilmann-Heimbach, Susanne Moebus, Bernhard G. Herrmann, Kristina Alsabeah, Carmen M. Burgos, Helene E. Lilja, Sahar Azodi, Pernilla Stenstrom, Einar Arnbjornsson, Barbora Frybova, Dariusz M. Lebensztejn, Wojciech Debek, Elwira Kolodziejczyk, Katarzyna Kozera, Jaroslaw Kierkus, Piotr Kalicinski, Marek Stefanowicz, Anna Socha-Banasiak, Michal Kolejwa, Anna Piaseczna-Piotrowska, Elzbieta Czkwianianc, Markus M. Noethen, Phillip Grote, Michal Rygl, Konrad Reinshagen, Nicole Spychalski, Barbara Ludwikowski, Jochen Hubertus, Andreas Heydweiller, Benno Ure, Oliver J. Muensterer, Ophelia Aubert, Jan-Hendrik Gosemann, Martin Lacher, Petra Degenhardt, Thomas M. Boemers, Anna Mokrowiecka, Ewa Malecka-Panas, Markus Woehr, Michael Knapp, Guido Seitz, Annelies de Klein, Grzegorz Oracz, Erwin Brosens, Heiko Reutter, Johannes Schumacher
Summary: This study identifies three risk loci and candidate genes associated with esophageal atresia with or without tracheoesophageal fistula (EA/TEF) through a genome-wide association study (GWAS), providing insights into the genetic architecture of EA/TEF.
HUMAN GENETICS AND GENOMICS ADVANCES
(2022)
Review
Surgery
Marek Szymczak, Piotr J. Kalicinski, Grzegorz Kowalewski, Mateusz Ciopinski, Malgorzata Markiewicz-Kijewska, Dorota Broniszczak, Bozenna Dembowska-Baginska, Andrzej Kosciesza, Grazyna Brzezinska-Rajszys, Waldemar Patkowski, Marek Stefanowicz
Summary: This report presents technical challenges and solutions in the reconstruction of the inferior vena cava and graft venous outflow during living donor liver transplantation in children. It concludes that reconstruction of multiple hepatic veins of living donor allografts can usually be successfully done with local venoplasty, while using cold-stored vein grafts may be beneficial in selected cases.
ANNALS OF TRANSPLANTATION
(2021)