Örnolfsson Kristjan T, Lund Sigrun H, Olafsson Sigurdur, Bergmann Ottar M, Björnsson Einar S
a Faculty of Medicine , University of Iceland, Reykjavík, Iceland.
b Division of Gastroenterology and Hepatology , Landspitali The National University Hospital of Iceland , Reykjavík , Iceland.
Scand J Gastroenterol. 2019 May;54(5):609-616. doi: 10.1080/00365521.2019.1606931. Epub 2019 May 10.
To assess the proportion of PBC patients with a biochemical response to ursodeoxycholic acid (UDCA) in a population-based cohort and the association of biochemical response with outcomes. All patients diagnosed with PBC in Iceland from 1991-2015 were identified. Patients taking UDCA for an adequate period of time were analyzed for treatment response according to the Barcelona, Paris I, Paris II and Toronto criteria and outcomes. Overall 182 females and 40 males were diagnosed with PBC and 135 patients were treated with UDCA. Overall 99 (73%) patients had adequate data on UDCA treatment and results of liver tests to assess biochemical response according to the Barcelona criteria, 95 (70%) according to the Toronto criterion and 85 (63%) according to the Paris I and II criteria. In all 74% ( = 63), 67% ( = 64), 54% ( = 53) and 46% ( = 39) responded to treatment according to the Paris I, Toronto, Barcelona and Paris II criteria. Among nonresponders according to the Paris I, Toronto, Paris II and Barcelona criteria, 50%, 39%, 33% and 30% developed cirrhosis versus 10%, 6%, 5% and 11% of responders, HR 5.36 ( = .002), 6.61 ( = .002), 10.94 ( = .003) and 2.21( = .11), respectively. Age-adjusted mortality was significantly lower among responders according to the Paris I and Paris II criteria, HR 0.33 ( = .02) and 0.31 ( = .02), respectively. Development of cirrhosis and higher mortality was significantly associated with a lack of biochemical response to UDCA. Frequent development of cirrhosis and increased mortality in nonresponders underlines the need for a more effective therapy than UDCA for this sizeable subgroup of patients.
评估基于人群队列中对熊去氧胆酸（UDCA）有生化反应的原发性胆汁性胆管炎（PBC）患者的比例，以及生化反应与预后的关联。确定了1991年至2015年在冰岛被诊断为PBC的所有患者。根据巴塞罗那、巴黎I、巴黎II和多伦多标准及预后情况，对服用UDCA足够时长的患者进行治疗反应分析。总体上，182名女性和40名男性被诊断为PBC，135名患者接受了UDCA治疗。总体而言，99名（73%）患者有关于UDCA治疗及肝脏检测结果的充分数据，可根据巴塞罗那标准评估生化反应；95名（70%）患者可根据多伦多标准评估；85名（63%）患者可根据巴黎I和巴黎II标准评估。根据巴黎I、多伦多、巴塞罗那和巴黎II标准，分别有74%（n = 63）、67%（n = 64）、54%（n = 53）和46%（n = 39）的患者对治疗有反应。在根据巴黎I、多伦多、巴黎II和巴塞罗那标准无反应的患者中，分别有50%、39%、33%和30%发展为肝硬化，而有反应的患者中这一比例分别为10%、6%、5%和11%，风险比分别为5.36（P = 0.002）、6.61（P = 0.002）、10.94（P = 0.003）和2.21（P = 0.11）。根据巴黎I和巴黎II标准，有反应患者的年龄调整死亡率显著较低，风险比分别为0.33（P = 0.02）和0.31（P = 0.02）。肝硬化的发展和较高的死亡率与对UDCA缺乏生化反应显著相关。无反应患者中肝硬化的频繁发生和死亡率的增加凸显了对于这一相当大的患者亚组，需要一种比UDCA更有效的治疗方法。
