There are limited drug options in the field of primary biliary cholangitis, so there is a great clinical need. In recent years, research and development of PBC treatment medications have been active domestically and internationally, and clinical trials have been conducted on multiple drugs with distinct targets. Therefore, on February 13, 2023, the State Drug Administration issued the \"Technical Guidelines for Clinical Trials of Drugs for the Treatment of Primary Biliary Cholangitis\" in order to guide and standardize the clinical trials of drugs for the treatment of PBC. This article briefly summarizes the key points of the guiding principles, focuses on the difficulties of clinical evaluation of drugs, discusses the key elements of clinical trials such as the selection of test populations and efficacy endpoints, and introduces the determination process through literature searches and expert discussion methods combined with reviewer experience and scientific considerations.
原发性胆汁性胆管炎（PBC）治疗领域药物选择有限，存在较大的临床需求。近年来，国内外PBC治疗药物研发活跃，有多个不同靶点的药物正在开展临床试验。为指导和规范PBC治疗药物临床试验,国家药品监督管理局于2023年2月13日发布了《原发性胆汁性胆管炎治疗药物临床试验技术指导原则》。现简要介绍指导原则的要点，围绕药物临床评价难点，通过文献检索和组织专家讨论等方法，结合审评经验，探讨了受试人群和有效性终点选择等临床试验关键要素，介绍了其确定过程和科学考量。.

In 2015, the Chinese Society of Hepatology and the Chinese Society of Gastroenterology published a consensus on primary biliary cholangitis (PBC). In the past years, numerous clinical studies have been published in the field of PBC. To guide the clinical diagnosis and management of PBC patients, the Chinese Society of Hepatology invited a panel of experts to assess the new clinical evidence and formulate the current guidelines.

暂无摘要。

The diagnosis of primary biliary cholangitis (PBC), an uncommon immune-mediated cholestatic liver disease, is based on positive circulating anti-mitochondrial (AMA) and/or PBC-specific anti-nuclear autoantibodies (ANA), coupled with elevated serum alkaline phopsphatase (ALP) levels. Timely initiation of treatment with ursodeoxycholic acid prevents progression to cirrhosis and liver failure. We aimed at investigating liver histology in patients with normal ALP level and positive AMA and/or PBC-specific ANA.
We searched the Swiss PBC Cohort Study database, which includes subjects with positive PBC autoimmune serology and normal ALP levels, for patients who underwent a liver biopsy. Histological slides were centrally reviewed by an expert liver pathologist, and sera were centrally re-tested for AMA and ANA.
30 patients were included; 90% females, median age 53 (range 27-72) years. Twenty-four (80%) had liver histology typical for (n = 2), consistent with (n = 16) or suggestive of (n = 6) PBC, including three of four AMA-negative ANA-positive patients. Among 22 ursodeoxycholic acid treated patients, 14 had elevated GGT levels before treatment; a significant decrease of the median GGT level between pre- (1.46 x ULN) and post- (0.43 x ULN) treatment (p = 0.0018) was observed.
In our series, a high proportion of AMA positive patients with normal ALP levels have PBC. For the first time we show histological diagnosis of PBC in AMA-negative/PBC-specific ANA-positive subjects and the potential role of GGT as a biomarker in PBC patients with normal baseline ALP levels. Current guidelines for the diagnosis of PBC do not cover the whole extent of PBC presentation, with important clinical implications in terms of timely treatment initiation.

Primary biliary cholangitis (PBC) is a chronic autoimmune cholestatic disease and may progress to liver fibrosis, liver cirrhosis, decompensated cirrhosis, and even end-stage liver disease without effective treatment. The diagnosis of PBC is mainly based on the biochemical parameters indicating cholestatic hepatitis and the presence of specific autoantibody in circulation. The goals of the treatment and management of PBC are to prevent the development of end-stage liver disease, to improve related clinical symptoms, and to improve patients\' quality of life. Since PBC has relatively strong heterogeneity and the clinical manifestations and course of PBC can be diverse, it is necessary to provide long-term individualized treatment and follow-up for such patients. Here we provide an interpretation of the 2017 EASL Clinical Practice Guidelines for the diagnosis and management of patients with PBC, in order to better understand recent clinical research evidence and updated recommendations. In particular, we focus on the key points in the diagnosis, treatment, and follow-up strategies of PBC and emphasizing that timely and accurate risk stratification and proper clinical research enrollment may bring benefits to patients with refractory PBC.
原发性胆汁性胆管炎（PBC）是一种慢性自身免疫性的胆汁淤积性疾病，如不给予有效治疗则可进展为肝纤维化、肝硬化及失代偿期，甚至终末期肝病。该疾病的诊断主要基于提示胆汁淤积性肝炎的生物化学指标以及循环中出现的特异性自身抗体。PBC治疗管理的主要目的是预防终末期肝病的发生，并改善疾病相关的临床症状，提高患者的生活质量。由于PBC是一种相对异质性较强的疾病，患者的临床表现和经过可有较大差异，因此给予患者长期的个体化治疗和随访是非常重要的。现对2017欧洲肝脏病学会发布的PBC诊治指南进行解读，以更好地理解新近出现的重要临床研究证据以及更新的推荐意见。尤其关注针对PBC的诊断、治疗及随访方面向临床提出的关键点，并强调及时准确地风险分层并将患者及时入组至恰当的临床研究可使难治性PBC患者受益。.