美国胃肠病协会（AGA）/美国肝病研究协会（AASLD）原发性胆汁性胆管炎管理临床实践指南。
完整摘要 · 中文译文
背景与目的：原发性胆汁性胆管炎（PBC）是一种慢性胆汁淤积性肝病，与进行性纤维化、肝硬化、肝移植和过早死亡相关。尽管已有有效疗法，但PBC的诊断和管理仍存在重要挑战，包括识别抗线粒体抗体阴性的疾病、评估对熊去氧胆酸（UDCA）的治疗应答、风险分层、应答不佳患者的治疗、胆汁淤积性瘙痒的管理，以及肝移植后疾病复发的预防。本指南旨在针对这些临床诊疗的关键领域制定循证建议。 方法：美国胃肠病学会（AGA）和美国肝病研究学会（AASLD）召集了一个多学科专家组，为成人PBC患者制定临床实践建议。对6个优先临床问题进行了系统回顾。采用推荐分级的评估、制定与评价（GRADE）框架对临床结局进行优先排序并评估证据，并利用其“证据到决策”框架制定建议。 结果：指南专家组就6条建议达成共识。这些建议涉及在疑似PBC但抗线粒体抗体阴性的患者中使用PBC特异性抗核抗体、推荐UDCA作为一线治疗、应用肝脏硬度测量进行预后判断，以及对UDCA应答不佳或不耐受的患者使用过氧化物酶体增殖物激活受体激动剂。 DCA、用于中重度瘙痒的linerixibat，以及移植后预防疾病复发的UDCA。证据的确定性从中等到极低不等。 结论：该指南强调早期诊断、优化一线治疗、基于风险的治疗升级、症状管理以及移植后疾病复发的预防，以改善患者预后和生活质量。
完整摘要 · 英文原文
BACKGROUND & AIMS: Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease associated with progressive fibrosis, cirrhosis, liver transplantation, and premature mortality. Despite effective therapies, important challenges remain in the diagnosis and management of PBC, including identification of antimitochondrial antibody-negative disease, assessment of response to ursodeoxycholic acid (UDCA), risk stratification, treatment of patients with inadequate response, management of cholestatic pruritus, and prevention of recurrent disease after liver transplantation. The aim of this guideline was to develop evidence-based recommendations addressing these key areas of clinical care. METHODS: The American Gastroenterological Association (AGA) and the American Association for the Study of Liver Diseases (AASLD) convened a multidisciplinary panel to develop clinical practice recommendations for adults with PBC. Systematic reviews were performed for 6 prioritized clinical questions. The Grading of Recommendations Assessment, Development and Evaluation framework was used to prioritize clinical outcomes and assess evidence, and its Evidence to Decision Framework was used to develop recommendations. RESULTS: The guideline panel agreed on 6 recommendations. These recommendations address the use of PBC-specific antinuclear antibodies in antimitochondrial antibody-negative suspected PBC, UDCA as first-line therapy, liver stiffness measurement for prognostication, peroxisome proliferator-activated receptor agonists for patients with inadequate response or intolerance to UDCA, linerixibat for moderate to severe pruritus, and UDCA prophylaxis post-transplantation to reduce disease recurrence. The certainty of evidence ranged from moderate to very low. CONCLUSIONS: The guideline emphasizes early diagnosis, optimization of first-line therapy, risk-based treatment escalation, symptom management, and prevention of post-transplantation disease recurrence to improve patient outcomes and quality of life.