Sohal Aalam, Kowdley Kris V
Department of Hepatology, Liver Institute Northwest, Seattle, WA, USA.
Department of Gastroenterology and Hepatology, Elson Floyd College of Medicine, Spokane, WA, USA.
Hepat Med. 2023 Jun 8;15:63-77. doi: 10.2147/HMER.S361077. eCollection 2023.
Primary biliary cholangitis (PBC), previously referred to as primary biliary cirrhosis, is an autoimmune disorder leading to the destruction of intra-hepatic bile ducts. If untreated, progressive bile duct damage and cholestasis can lead to ductopenia and result in cirrhosis. Ursodiol, the first drug approved for PBC, has changed the natural history of this disease and improved patient outcomes. Subsequently, several new prediction models incorporating a response to ursodiol were developed. These include the GLOBE score, which was shown to predict long-term outcomes in patients with PBC. In 2016, obeticholic acid (OCA) became the second drug to be approved by the FDA, predominantly based on improvement in alkaline phosphatase (ALP) levels. This trial has subsequently influenced the design of clinical trials. Several drugs are currently being evaluated as therapeutic options for PBC, with improvement in ALP being a main endpoint. In this review, we will discuss the impact of new therapies on GLOBE scores in patients with PBC.
原发性胆汁性胆管炎（PBC），以前称为原发性胆汁性肝硬化，是一种自身免疫性疾病，可导致肝内胆管破坏。如果不进行治疗，进行性胆管损伤和胆汁淤积可导致小胆管减少并导致肝硬化。熊去氧胆酸是首个被批准用于治疗PBC的药物，它改变了这种疾病的自然病程并改善了患者的预后。随后，开发了几种纳入对熊去氧胆酸反应的新预测模型。其中包括GLOBE评分，该评分被证明可预测PBC患者的长期预后。2016年，奥贝胆酸（OCA）成为第二种获得美国食品药品监督管理局（FDA）批准的药物，主要基于碱性磷酸酶（ALP）水平的改善。该试验随后影响了临床试验的设计。目前正在评估几种药物作为PBC的治疗选择，以ALP的改善作为主要终点。在本综述中，我们将讨论新疗法对PBC患者GLOBE评分的影响。
