Abstract
BackgroundPolycystic 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/designThis 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.DiscussionThis 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.Trial registration numberClinicalTrials.gov: NCT01157858
Highlights
Polycystic liver disease (PLD) is defined as having more than 20 liver cysts and can present as a severe and disabling condition
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
Summary
The study has been submitted to clinicaltrials.gov and the trial identifier is NCT01157858. All continuous variables are stated as mean (SD) All PCLD patients underwent mutation analysis, no mutation analysis was performed on ADPKD patients There were no differences between the treatment arms lanreotide reduced PLD volume by 2.9% when compared to baseline values [7]. After 18 months of treatment, there was no difference in kidney cyst growth between the groups [20] Another group studied everolimus in 433 ADPKD patients in a 2-year, double-blind trial. Sirolimus given for an average of 19 months decreased PLD volume by 11.9%, whereas tacrolimus for a comparable duration increased PLD volume by 14.1% [10] These data led to the assumption that a large effect size of everolimus treatment is realistic. The sponsor had no role in the conduct of the study; the collection, management, analysis, or interpretation of data; or in the preparation, review, or approval of the manuscript
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