Department of Medical and Surgical Sciences, Alma Mater Studiorum - University of Bologna, Azienda Ospedaliera Universitaria Sant'Orsola-Malpighi, Bologna, Italy.
Liver Int. 2015 Feb;35(2):636-41. doi: 10.1111/liv.12560. Epub 2014 Apr 23.
BACKGROUND & AIMS: In recent years, primary biliary cirrhosis is mostly diagnosed in patients who are asymptomatic; however, a proportion of cases still present with typical complaints such as fatigue and/or pruritus. We compared biochemical, histological and immunological features of patients with or without fatigue and/or pruritus at onset to see whether the different clinical presentation may eventually impact on disease progression.
We analysed the Bologna cohort of 216 patients with primary biliary cirrhosis referred to our Centre between 1997 and 2007, according to symptomatic (fatigue and/or pruritus) or asymptomatic presentation. Clinical, biochemical, histological and immunological feature at diagnosis, response to ursodeoxycholic acid and progression of the disorder were compared after a mean follow-up of 81 ± 75 months.
At diagnosis, symptomatic patients were significantly more often women (98.6% vs. 87.2%, P = 0.004), younger (mean age 49 ± 12 vs. 55 ± 12 years, P = 0.003) and with more pronounced biochemical activity, as indicated by higher alkaline phosphatase (mean 2.93 ± 2 vs. 2.12, P = 0.002) and aminotransferase (mean 1.92 ± 1 vs. 1.47 ± 1.27, P = 0.014) levels, whereas histological stage and autoantibody profile were similar. Symptomatic patients were less likely to respond to ursodeoxycholic acid therapy (63% vs. 81%, P = 0.006) and developed more often cirrhosis and its complications (31% vs. 13%, P = 0.004).
Fatigue and/or pruritus at onset identify a subset of patients with primary biliary cirrhosis who preferentially are women, younger, with a particularly active disease, less responsive to ursodeoxycholic acid treatment, and more inclined to evolve to cirrhosis and its complications.
近年来，原发性胆汁性肝硬化多在无症状患者中诊断；然而，仍有一部分病例表现为典型的疲劳和/或瘙痒症状。我们比较了有或无疲劳和/或瘙痒症状的患者的生化、组织学和免疫学特征，以了解不同的临床表现是否最终会影响疾病的进展。
我们根据症状（疲劳和/或瘙痒）或无症状表现，分析了 1997 年至 2007 年间我们中心收治的 216 例原发性胆汁性肝硬化患者的博洛尼亚队列。在平均随访 81±75 个月后，比较了诊断时的临床、生化、组织学和免疫学特征、对熊去氧胆酸的反应以及疾病的进展情况。
在诊断时，有症状的患者中女性（98.6% vs. 87.2%，P=0.004）、年龄较小（平均年龄 49±12 岁 vs. 55±12 岁，P=0.003）和生化活性较高的患者明显更多，表现为碱性磷酸酶（平均 2.93±2 vs. 2.12，P=0.002）和氨基转移酶（平均 1.92±1 vs. 1.47±1.27，P=0.014）水平较高，而组织学分期和自身抗体谱相似。有症状的患者对熊去氧胆酸治疗的反应性较低（63% vs. 81%，P=0.006），更容易发展为肝硬化及其并发症（31% vs. 13%，P=0.004）。
发病时的疲劳和/或瘙痒可识别出原发性胆汁性肝硬化患者的一个亚组，这些患者更倾向于是女性、年轻、疾病活动度特别高、对熊去氧胆酸治疗反应较差、更倾向于发展为肝硬化及其并发症。
