Nousia-Arvanitakis S, Fotoulaki M, Economou H, Xefteri M, Galli-Tsinopoulou A
Fourth Department of Pediatrics, Aristotle University of Thessaloniki, Greece.
J Clin Gastroenterol. 2001 Apr;32(4):324-8. doi: 10.1097/00004836-200104000-00009.
To evaluate the efficacy of UDCA in arresting the progression of the early multifocal hepatic lesion to overt CF-related NBC.
Focal biliary cirrhosis is an early hepatic pathologic change related to the ion transport defect in cystic fibrosis. The factors involved in the progression of focal to nodular biliary cirrhosis are not clear. Ursodeoxycholic--a hydrophilic, nontoxic, choleretic, and hepatoprotective exogenous bile acid--has been reported to be effective in the management of cholestatic liver disease.
For 10 years at 6-month intervals, 70 individuals with cystic fibrosis (38 men and 32 women; age range, 2--29 years) were examined using hepatosplenomegaly, liver function tests, and ultrasound liver scan. Patients demonstrating evidence of liver involvement at the onset or during the study received ursodeoxycholic acid 20 mg/kg body weight.
After the administration of ursodeoxycholic acid, the progression of nodular biliary cirrhosis ultrasound changes was arrested, hepatic function was preserved, and no variceal bleeding was observed. No case of focal biliary cirrhosis progressed to nodular biliary cirrhosis. Furthermore, the multifocal, multilobular changes suggestive of focal biliary cirrhosis on ultrasound scan were reversed to normal.
Ursodeoxycholic acid is effective in improving cholestasis and hepatic dysfunction in nodular biliary cirrhosis and, also, in reversing the early sonography findings suggestive of focal biliary cirrhosis. It is speculated that ursodeoxycholic acid may prove to affect the natural history of cystic fibrosis-related liver disease.
评估熊去氧胆酸（UDCA）阻止早期多灶性肝损伤进展为明显的囊性纤维化（CF）相关的结节性胆汁性肝硬化（NBC）的疗效。
局灶性胆汁性肝硬化是一种与囊性纤维化中离子转运缺陷相关的早期肝脏病理变化。局灶性胆汁性肝硬化进展为结节性胆汁性肝硬化的相关因素尚不清楚。熊去氧胆酸——一种亲水性、无毒、利胆且具有肝保护作用的外源性胆汁酸——已被报道在胆汁淤积性肝病的治疗中有效。
在10年的时间里，每隔6个月对70例囊性纤维化患者（38名男性和32名女性；年龄范围为2至29岁）进行肝脾肿大、肝功能检查和肝脏超声扫描。在研究开始时或研究期间有肝脏受累证据的患者接受20mg/kg体重的熊去氧胆酸治疗。
给予熊去氧胆酸后，结节性胆汁性肝硬化的超声变化进展得到阻止，肝功能得以保留，且未观察到静脉曲张出血。没有局灶性胆汁性肝硬化进展为结节性胆汁性肝硬化的病例。此外，超声扫描显示的提示局灶性胆汁性肝硬化的多灶性、多叶性变化恢复正常。
熊去氧胆酸在改善结节性胆汁性肝硬化的胆汁淤积和肝功能障碍方面有效，并且在逆转提示局灶性胆汁性肝硬化的早期超声检查结果方面也有效。据推测，熊去氧胆酸可能会影响囊性纤维化相关肝病的自然病程。
