Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands.
Trials. 2011 Nov 21;12:246. doi: 10.1186/1745-6215-12-246.
Polycystic liver disease (PLD) is defined as having more than 20 liver cysts and can present as a severe and disabling condition. Most symptoms are caused by the mass effect of the liver size and include abdominal pain and distension. The somatostatin analogues octreotide and lanreotide have proven to reduce polycystic liver volume. mTOR inhibitors such as everolimus inhibit cell proliferation and might thereby reduce growth of liver cysts. This trial aims to assess the benefit of combination therapy of everolimus and octreotide compared to octreotide monotherapy. In this study we present the structure of the trial and the characteristics of the included patients.
METHODS/DESIGN: This is a randomized open-label clinical trial comparing the effect of 12 months of everolimus and octreotide to octreotide monotherapy in PLD patients. Primary outcome is change in liver volume determined by CT-volumetry. Secondary outcomes are changes in abdominal symptoms and quality of life. Moreover, safety and tolerability of the drugs will be assessed.
This trial will compare the relative efficacy of combination therapy with octreotide and everolimus to octreotide monotherapy. Since they apply to different pathways of cystogenesis we expect that combining octreotide and everolimus will result in a cumulative reduction of polycystic liver volume.
ClinicalTrials.gov: NCT01157858.
多囊肝疾病（PLD）定义为肝脏内存在超过 20 个囊肿，可表现为严重且致残的疾病。大多数症状是由肝脏大小的占位效应引起的，包括腹痛和腹胀。生长抑素类似物奥曲肽和兰瑞肽已被证明可减少多囊肝的体积。mTOR 抑制剂如依维莫司可抑制细胞增殖，从而可能减少肝囊肿的生长。本试验旨在评估依维莫司和奥曲肽联合治疗与奥曲肽单药治疗的益处。在这项研究中，我们介绍了试验的结构和纳入患者的特征。
方法/设计：这是一项随机、开放标签的临床试验，比较了依维莫司和奥曲肽联合治疗与奥曲肽单药治疗在 PLD 患者中的疗效。主要结局是通过 CT 体积测量法确定的肝体积变化。次要结局是腹部症状和生活质量的变化。此外，还将评估药物的安全性和耐受性。
本试验将比较奥曲肽和依维莫司联合治疗与奥曲肽单药治疗的相对疗效。由于它们作用于不同的囊肿形成途径，我们预计奥曲肽和依维莫司联合治疗将导致多囊肝体积的累积减少。
ClinicalTrials.gov：NCT01157858。
