PILOT STUDY OF BUDESONIDE FOR PATIENTS WITH PSC AND PBC

原发性硬化性胆管炎和原发性胆汁性肝硬变 成人慢性肝病的重要原因。这两个都是 疾病是进行性的，可能需要原位肝移植。 用于管理末期阶段。目前还没有有效的治疗方法 原发性硬化性胆管炎。熊去氧胆酸已经证明 明确部分原发性胆道疾病患者的临床益处 但不幸的是，相当数量的患者患有 原发性胆汁性肝硬变似乎对 熊去氧胆酸。正因为如此，需要额外的 两组患者的治疗形式，识别 治疗是我们的长期目标。皮质类固醇在这些药物中可能有价值 患者，有限的信息支持皮质类固醇的使用 在这些患者群体中。对与胆汁淤积相关的担忧 骨质疏松症使临床医生对使用 对这些患者使用常规的皮质类固醇。最近，布地奈德， 一种新的皮质类固醇具有广泛的首过肝代谢和 据推测，已经出现了最小的系统性副作用。这 从理论上讲，药物可以提供常规药物的潜在好处 没有相关副作用的皮质类固醇。这项初步研究 将评估布地奈德治疗慢性阻塞性肺疾病的安全性和有效性 原发性硬化性胆管炎和原发性胆汁性肝硬变 对熊去氧胆酸的生化反应不佳的患者。 这将使我们能够估计其疗效和安全性。 布地奈德在这项研究中，我们密切监测了 布地奈德在这项研究中，我们密切监测了 胆脂素相关性骨质疏松症的布地奈德。简单的两个阶段 设计将用于最少30名患者，最多50名患者 入选患者(15或25名患有原发性硬化性胆管炎的患者 和15或25例原发性胆汁性肝硬变)。审判将会 根据结果，分两到三年进行 第一阶段。如果这种药物看起来很有希望，而且耐受性很好， 将计划进行随机试验。

Primary sclerosing cholangitis and primary-biliary cirrhosis are important causes of chronic liver disease in adults. Both of these diseases are progressive and may require orthotopic liver transplantation for the management of the end stages. There is no effective therapy for primary sclerosing cholangitis. Ursodeoxycholic acid has demonstrated clearcut clinical benefit in some patients with primary biliary cirrhosis, but unfortunately, a substantial number of patients with primary biliary cirrhosis appear to have a suboptimal response to ursodeoxycholic acid. Because of this, a need exists for additional forms of therapy in both groups of patients, identification of such therapy is our long-term goal. Corticosteriods may be of value in these patients, and limited information supports the use of corticosteroids in these groups of patients. Concerns about cholestasis-associated osteoporosis have made clinicians quite cautious about the use of conventional corticosteroids for these patients. Recently,, budesonide, a new corticosteroid with extensive first pass hepatic metabolism and presumably minimal systemic side effects has become available. This drug, in theory, could provide the potential benefits of conventional corticosteroids without the associated side effects. This pilot study will assess the safety and efficacy of budesonide in patients with primary sclerosing cholangitis as well as primary billiary cirrhosis patients with suboptimal biochemical response to ursodeoxychoic acid. This will allow us to estimate the efficacy and assess the safety of budesonide during this study as we closely monitor the effects of budesonide during this study as we closely monitor the effects of budesonide of cholestestatis-associated osteoporosis. A simple two-stage design will be used with a minimum of 30 patients and a maximum of 50 patients entered (15 or 25 patients with primary sclerosing cholangitis and 15 or 25 patients with primary billary cirrhosis). The trial will be conducted over a two-to three-year period, depending on the results of the first stage. If the drug looks promising and is well tolerated, a randomized trial will be planned.