title: Long-term Effects of Octreotide on Liver Volume in Patients With Polycystic Kidney and Liver Disease.
abstract: Short-term studies have shown that somatostatin analogues are effective in patients with polycystic kidney and liver disease. We evaluated the long-term effects of long-acting release octreotide (octreotide LAR), a somatostatin inhibitor, vs placebo in these patients.
  
  
  
  We performed a controlled study of adults with polycystic kidney and liver disease (estimated glomerular filtration rate, 40 mL/min/1.73m(2) or more) at a single center in Italy. We analyzed data from 27 patients randomly assigned to groups given octreotide LAR (40 mg, n = 14) or placebo (n = 13) each month for 3 years. The primary outcome was absolute and percentage change in total liver volume (TLV), which was measured by magnetic resonance imaging at baseline, after 3 years of treatment, and then 2 years after treatment ended.
  
  
  
  Baseline characteristics were similar between groups. After 3 years, TLV decreased by 130.2 ± 133.2 mL in patients given octreotide LAR (7.8% ± 7.4%) (P = .003) but increased by 144.3 ± 316.8 mL (6.1% ± 14.1%) in patients given placebo. Change vs baseline differed significantly between groups (P = .004). Two years after treatment ended, TLV had decreased 14.4 ± 138.4 mL (0.8% ± 9.7%) from baseline in patients given octreotide LAR but increased by 224.4 ± 331.7 mL (11.0% ± 14.4%) in patients given placebo. Changes vs baseline still differed significantly between groups (P = .046). Decreases in TLV were similar in each sex; the change in TLV was greatest among subjects with larger baseline TLV. No patient withdrew because of side effects.
  
  
  
  In a placebo-controlled study of patients with polycystic kidney and liver disease, 3 years of treatment with octreotide LAR significantly reduced liver volume; reductions were maintained for 2 years after treatment ended. Octreotide LAR was well-tolerated. ClinicalTrials.gov number: NCT02119052.
authors: Nephrology Unit, Department of Public Health, Federico II University, Naples, Italy.
authors: Antonio
authors: A
authors: Pisani
authors: Nephrology Unit, Department of Public Health, Federico II University, Naples, Italy.
authors: Massimo
authors: M
authors: Sabbatini
authors: Department of Radiology, Federico II University, Naples, Italy.
authors: Massimo
authors: M
authors: Imbriaco
authors: Nephrology Unit, Department of Public Health, Federico II University, Naples, Italy.
authors: Eleonora
authors: E
authors: Riccio
authors: IRCCS - Istituto di Ricerche Farmacologiche "Mario Negri", Clinical Research Centre for Rare Diseases "Aldo e Cele Daccò", Bergamo, Italy.
authors: Nadia
authors: N
authors: Rubis
authors: Institute of Biostructure and Bioimaging, National Research Council, Naples, Italy.
authors: Anna
authors: A
authors: Prinster
authors: IRCCS - Istituto di Ricerche Farmacologiche "Mario Negri", Clinical Research Centre for Rare Diseases "Aldo e Cele Daccò", Bergamo, Italy.
authors: Annalisa
authors: A
authors: Perna
authors: Institute of Biostructure and Bioimaging, National Research Council, Naples, Italy.
authors: Raffaele
authors: R
authors: Liuzzi
authors: Department of Advanced Biomedical Sciences, Federico II University, Naples, Italy.
authors: Letizia
authors: L
authors: Spinelli
authors: Department of Surgical Sciences and Nephrology, Federico II University, Naples, Italy.
authors: Michele
authors: M
authors: Santangelo
authors: IRCCS - Istituto di Ricerche Farmacologiche "Mario Negri", Clinical Research Centre for Rare Diseases "Aldo e Cele Daccò", Bergamo, Italy; Nephrology and Dialysis Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy; Department of Biomedical and Clinical Sciences, University of Milan, Milan, Italy. Electronic address: Giuseppe.remuzzi@marionegri.it.
authors: Giuseppe
authors: G
authors: Remuzzi
authors: IRCCS - Istituto di Ricerche Farmacologiche "Mario Negri", Clinical Research Centre for Rare Diseases "Aldo e Cele Daccò", Bergamo, Italy; Nephrology and Dialysis Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
authors: Piero
authors: P
authors: Ruggenenti
mesh_terms: D000328:Adult; D003560:Cysts / Q000188:drug therapy*; D005260:Female; D005765:Gastrointestinal Agents / Q000627:therapeutic use*; D006801:Humans; D007558:Italy; D008099:Liver / Q000000981:diagnostic imaging / Q000187:drug effects* / Q000473:pathology*; D008107:Liver Diseases / Q000188:drug therapy*; D008279:Magnetic Resonance Imaging; D008297:Male; D015282:Octreotide / Q000627:therapeutic use*; D010919:Placebos / Q000008:administration & dosage; D016891:Polycystic Kidney, Autosomal Dominant / Q000150:complications*; D011446:Prospective Studies; D016037:Single-Blind Method; D013995:Time; D016896:Treatment Outcome
chemical_list: D005765:Gastrointestinal Agents; D010919:Placebos; D015282:Octreotide
keywords: ADPKD Volume; Cyst Growth; Somatostatin Analogue