Alomari Mohammad, Covut Fahrettin, Al Momani Laith, Chadalavada Pravallika, Hitawala Asif, Young Mark F, Romero-Marrero Carlos
Department of Internal Medicine Cleveland Clinic Foundation Cleveland Ohio USA.
Department of Internal Medicine East Tennessee State University Johnson City Tennessee USA.
JGH Open. 2019 Jul 22;4(2):132-139. doi: 10.1002/jgh3.12223. eCollection 2020 Apr.
The United Kingdom-primary biliary cholangitis (UK-PBC) and global primary biliary cholangitis group (GLOBE) prognostic models have been recently developed to predict long-term outcomes in primary biliary cholangitis (PBC). However, these predictive scores have not yet been well evaluated in the U.S. population.
We retrospectively reviewed newly diagnosed PBC patients at the Cleveland Clinic between November 1998 and February 2017. Adverse events were defined as liver transplantation, liver-related mortality, and all-cause mortality. Transplant-free survival (TFS) was estimated using the Kaplan-Meier method. Predictive performances of all prognostic models were evaluated using the C-statistic.
We identified 352 patients who used ursodeoxycholic acid therapy. Of them, 311 (88.4%) only had PBC, while 41 (11.6%) were diagnosed with PBC-autoimmune hepatitis overlap. A total of 22 (6%), 47 (13%), and 55 (16%) patients had adverse events within 5, 10, and 15 years after diagnosis, respectively. In patients with PBC only, the C-statistic in predicting 15-year adverse events was 0.75 per GLOBE compared to 0.74 per UK-PBC ( = 0.94), 0.73 per Rotterdam ( = 0.44), 0.66 per Barcelona ( = 0.004), 0.65 per Paris 1 ( = 0.005), 0.62 per Paris 2 ( < 0.0001), 0.60 per Toronto ( < 0.0001), and 0.60 per Mayo ( < 0.0001) scores. Median follow-up was 9.2 years. Ten-year TFS for patients who had optimal suboptimal treatment response was 92 74% per Paris 1 ( < 0.0001), 95 79% per Paris 2 ( = 0.0002), 93 65% per Barcelona ( < 0.0001), and 96 68% per Rotterdam ( < 0.0001) risk scores, respectively.
In our cohort of PBC patients, the UK-PBC and GLOBE scores were both accurate and reasonably valid prognostic models in the U.S. population.
英国原发性胆汁性胆管炎（UK-PBC）和全球原发性胆汁性胆管炎组（GLOBE）预后模型最近已被开发出来，用于预测原发性胆汁性胆管炎（PBC）的长期预后。然而，这些预测评分在美国人群中尚未得到充分评估。
我们回顾性分析了1998年11月至2017年2月在克利夫兰诊所新诊断的PBC患者。不良事件定义为肝移植、肝脏相关死亡率和全因死亡率。采用Kaplan-Meier方法估计无移植生存期（TFS）。使用C统计量评估所有预后模型的预测性能。
我们确定了352例接受熊去氧胆酸治疗的患者。其中，311例（88.4%）仅患有PBC，而41例（11.6%）被诊断为PBC-自身免疫性肝炎重叠综合征。分别有22例（6%）、47例（13%）和55例（16%）患者在诊断后5年、10年和15年内发生不良事件。在仅患有PBC的患者中，预测15年不良事件的C统计量，GLOBE模型为0.75，UK-PBC模型为0.74（P = 0.94），鹿特丹模型为0.73（P = 0.44），巴塞罗那模型为0.66（P = 0.004），巴黎1模型为0.65（P = 0.005），巴黎2模型为0.62（P < 0.0001），多伦多模型为0.60（P < 0.0001），梅奥模型为0.60（P < 0.0001）。中位随访时间为9.2年。根据巴黎1风险评分，治疗反应最佳与次优的患者10年TFS分别为92%和7(4%)（P < 0.0001）；根据巴黎2风险评分，分别为95%和79%（P = 0.0002）；根据巴塞罗那风险评分，分别为93%和65%（P < 0.0001）；根据鹿特丹风险评分，分别为96%和68%（P < （此处原文可能有误，推测为P < 0.0001））。
在我们的PBC患者队列中，UK-PBC和GLOBE评分在美国人群中都是准确且合理有效的预后模型。
