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化是 成人慢性肝病的重要原因。 这两 疾病是进行性的，可能需要原位肝移植 管理最后阶段。 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 某些原发性胆道疾病患者的明确临床获益 肝硬化，但不幸的是，大量的患者 原发性胆汁性肝硬化似乎对 熊去氧胆酸 正因为如此，需要额外的 两组患者的治疗形式， 治疗是我们的长期目标 皮质类固醇可能是有价值的， 患者，有限的信息支持使用皮质类固醇 在这些患者中。对胆汁淤积相关的担忧 骨质疏松症使临床医生对使用 传统的皮质类固醇激素。 最近，布地奈德， 具有广泛首过肝代谢的新型皮质类固醇， 大概已经可以获得最小的全身副作用。 这 理论上，药物可以提供常规药物的潜在益处。 皮质类固醇而没有相关的副作用。 这项初步研究 将评估布地奈德在以下患者中的安全性和有效性： 原发性硬化性胆管炎以及原发性胆汁性肝硬化 对熊去氧胆酸生化反应不佳的患者。 这将使我们能够估计疗效和评估的安全性 在这项研究中，我们密切监测布地奈德的作用， 在这项研究中，我们密切监测布地奈德的作用， 布地奈德治疗阿替卡松相关骨质疏松症。 一个简单的两阶段 设计将用于最少30名患者和最多50名患者 入组患者（15或25例原发性硬化性胆管炎患者 和15或25例原发性胆汁性肝硬化患者）。 审判将 根据结果，在两到三年内进行 的第一阶段。 如果药物看起来有希望并且耐受性良好， 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.