J Pediatr Gastroenterol Nutr 2017 10;65(4):443-448
*Pediatric Hepatology Unit, APHP-Hôpital Necker and UPMC Univ Paris 06, INSERM, UMR_S 938, Centre de Recherche Saint-Antoine, Paris, France †Digestive Health Institute, Children's Hospital Colorado and Section of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of Colorado School of Medicine Aurora, CO ‡Pediatric Gastroenterology and Hepatology, Department Pediatrics, Beatrix Children's Hospital/University Medical Center Groningen, University of Groningen, Groningen, The Netherlands §CF Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy ||Sorbonne Universités, UPMC Univ Paris 06, INSERM, UMR_S 938, Centre de Recherche Saint-Antoine, Institute of Cardiometabolism and Nutrition, Institut Hospitalo-Universitaire & Assistance Publique-Hôpitaux de Paris, Hôpital Saint-Antoine, Centre de Référence Maladies Rares des Maladies Inflammatoires des Voies Biliaires, Service d'Hépatologie, Paris, France ¶Department of Gastroenterology & Hepatology, Erasmus University Medical Center, Rotterdam #Center for Liver, Digestive and Metabolic Diseases, Department of Pediatrics, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands **The Liver Unit, Birmingham Children's Hospital, Birmingham, UK ††Division of Gastroenterology, Hepatology & Nutrition, The Hospital for Sick Children, Department of Paediatrics, University of Toronto, Toronto, Ontario, Canada ‡‡Sorbonne Universités, UPMC Univ Paris 06, INSERM, UMR_S 938, Centre de Recherche Saint-Antoine, Institute of Cardiometabolism and Nutrition, Institut Hospitalo-Universitaire & Assistance Publique-Hôpitaux de Paris, Hôpital Pitié-Salpêtrière §§Paris-Descartes, Sorbonne Paris-Cité University, INSERM U-1223 - Pasteur Institute, and Hepatology Unit, Assistance Publique-Hôpitaux de Paris, Cochin Hospital, Paris, France ||||Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria ¶¶Department of Paediatric Gastroenterology, Hepatology and Nutrition University Hospitals Leuven, Leuven, Belgium ##Department of Paediatric Kidney, Liver and Metabolic Diseases, Division of Paediatric Gastroenterology and Hepatology, Hannover Medical School, Hannover, Germany ***Pediatric Gastroenterology, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Objectives: Hepatobiliary complications are a leading cause of morbidity and mortality in cystic fibrosis (CF) patients. Knowledge of the underlying pathological aspects and optimal clinical management is, however, sorely lacking.
Methods: We provide a summary of the lectures given by international speakers at the European Society for Paediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN) monothematic conference on cystic fibrosis-related liver disease (CFLD) held in Paris in January 2016, to discuss the status of our current knowledge of liver disease in CF patients, to define the critical areas that need to be addressed, and to resolve actions to elucidate relevant mechanisms of disease to optimise future therapeutic options.
Conclusions: The need for a universal consensus on the definition of CFLD to clarify disease stage and to identify relevant biomarkers to assess disease severity was highlighted. A deeper understanding of the pathophysiology and prognostic factors for the long-term evolution of CFLD is fundamental to move forward and has a strong bearing on identifying potential treatments. Novel experimental models and new treatment options under investigation are discussed and offer hope for the near future of CFLD.