title: Everolimus and long acting octreotide as a volume reducing treatment of polycystic livers (ELATE): study protocol for a randomized controlled trial.
abstract: BACKGROUND
  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
  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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  ClinicalTrials.gov: NCT01157858.
authors: Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands.
authors: Melissa
authors: M
authors: Chrispijn
authors: Joost P H
authors: JP
authors: Drenth
mesh_terms: D000328:Adult; D000368:Aged; D002985:Clinical Protocols; D003560:Cysts / Q000188:drug therapy*; D004359:Drug Therapy, Combination; D000068338:Everolimus; D006801:Humans; D008107:Liver Diseases / Q000188:drug therapy*; D008875:Middle Aged; D015282:Octreotide / Q000008:administration & dosage*; D018401:Sample Size; D020123:Sirolimus / Q000008:administration & dosage / Q000031:analogs & derivatives*; D014057:Tomography, X-Ray Computed
chemical_list: D000068338:Everolimus; D015282:Octreotide; D020123:Sirolimus