Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Stanford University, Palo Alto, California, USA.
Department of Medicine, Section of Digestive Diseases, Yale School of Medicine, New Haven, Connecticut, USA.
Hepatology. 2024 May 1;79(5):1220-1238. doi: 10.1097/HEP.0000000000000646. Epub 2023 Oct 26.
Cystic fibrosis (CF) may cause a spectrum of hepatobiliary complications, including portal hypertension, multilobular cirrhosis, and liver failure. Current guidelines on the detection and monitoring of hepatobiliary complications in CF were published in 1999. The CF Foundation assembled a committee to evaluate research advances and formulate revised guidelines for CF-associated liver disease. A committee of hepatologists, gastroenterologists, pulmonologists, pharmacists, nurses, dietitians, individuals with CF, and the parents of a child with CF devised "population, intervention, comparison, and outcome" questions regarding hepatobiliary disease in CF. PubMed literature searches were performed for each population, intervention, comparison, and outcome question. Recommendations were voted on with 80% agreement required to approve a recommendation. Public comment on initial recommendations was solicited prior to the formulation of final recommendations. Thirty-one population, intervention, comparison, and outcome questions were assembled, 6401 manuscripts were title screened for relevance, with 1053 manuscripts undergoing detailed full-text review. Seven recommendations were approved for screening, 13 for monitoring of existing disease, and 14 for treatment of CF-associated hepatobiliary involvement or advanced liver disease. One recommendation on liver biopsy did not meet the 80% threshold. One recommendation on screening ultrasound was revised and re-voted on. Through a multidisciplinary committee and public engagement, we have assembled updated recommendations and guidance on screening, monitoring, and treatment of CF-associated hepatobiliary involvement and advanced liver disease. While research gaps remain, we anticipate that these recommendations will lead to improvements in CF outcomes through earlier detection and increased evidence-based approaches to monitoring and treatment.
囊性纤维化 (CF) 可能导致一系列肝胆并发症，包括门静脉高压、多小叶性肝硬化和肝功能衰竭。1999 年发布了 CF 肝胆并发症检测和监测的现行指南。CF 基金会组织了一个委员会，以评估研究进展并制定 CF 相关肝病的修订指南。一组肝胆病专家、胃肠病专家、肺病专家、药剂师、护士、营养师、CF 患者以及 CF 患儿的家长制定了 CF 肝胆疾病的“人群、干预、比较和结局”问题。针对每个人群、干预、比较和结局问题，进行了 PubMed 文献检索。建议需要获得 80%的投票赞成才能获得批准。在制定最终建议之前，征求了公众对初步建议的意见。共提出了 31 个人群、干预、比较和结局问题，筛选出 6401 篇标题与相关性相关的文献，对 1053 篇文献进行了详细的全文审查。有 7 项建议被批准用于筛查，13 项用于监测现有疾病，14 项用于治疗 CF 相关的肝胆受累或晚期肝病。一项关于肝活检的建议未达到 80%的门槛。一项关于超声筛查的建议进行了修订并重新投票。通过多学科委员会和公众参与，我们已经制定了关于 CF 相关肝胆受累和晚期肝病筛查、监测和治疗的更新建议和指导。虽然仍存在研究空白，但我们预计这些建议将通过早期发现和增加基于证据的监测和治疗方法来改善 CF 结局。
