Debray Dominique, Narkewicz Michael R, Bodewes Frank A J A, Colombo Carla, Housset Chantal, de Jonge Hugo R, Jonker Johan W, Kelly Deirdre A, Ling Simon C, Poynard Thierry, Sogni Philippe, Trauner Michael, Witters Peter, Baumann Ulrich, Wilschanski Michael, Verkade Henkjan J
*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.
J Pediatr Gastroenterol Nutr. 2017 Oct;65(4):443-448. doi: 10.1097/MPG.0000000000001676.
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.
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.
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.
肝胆并发症是囊性纤维化（CF）患者发病和死亡的主要原因。然而，对于其潜在病理方面的认识以及最佳临床管理却极为匮乏。
我们总结了2016年1月在巴黎举行的欧洲儿科胃肠病学、肝病学和营养学会（ESPGHAN）关于囊性纤维化相关肝病（CFLD）的专题会议上国际演讲者的讲座内容，以讨论我们目前对CF患者肝病的认识状况，确定需要解决的关键领域，并确定阐明疾病相关机制以优化未来治疗选择的行动。
强调了对CFLD定义达成普遍共识以明确疾病阶段并识别评估疾病严重程度的相关生物标志物的必要性。深入了解CFLD长期演变的病理生理学和预后因素对于取得进展至关重要，并且对确定潜在治疗方法有重要影响。文中讨论了正在研究的新型实验模型和新的治疗选择，为CFLD的近期未来带来了希望。
