Antonio Pisani1, Massimo Sabbatini1, Massimo Imbriaco2, Eleonora Riccio1, Nadia Rubis3, Anna Prinster4, Annalisa Perna3, Raffaele Liuzzi4, Letizia Spinelli5, Michele Santangelo6, Giuseppe Remuzzi7, Piero Ruggenenti8. 1. Nephrology Unit, Department of Public Health, Federico II University, Naples, Italy. 2. Department of Radiology, Federico II University, Naples, Italy. 3. IRCCS - Istituto di Ricerche Farmacologiche "Mario Negri", Clinical Research Centre for Rare Diseases "Aldo e Cele Daccò", Bergamo, Italy. 4. Institute of Biostructure and Bioimaging, National Research Council, Naples, Italy. 5. Department of Advanced Biomedical Sciences, Federico II University, Naples, Italy. 6. Department of Surgical Sciences and Nephrology, Federico II University, Naples, Italy. 7. 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. 8. 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.
Abstract
BACKGROUND & AIMS: 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. METHODS: 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 givenoctreotide 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. RESULTS: 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. CONCLUSIONS: In a placebo-controlled study of patients with polycystic kidney and liver disease, 3 years of treatment withoctreotide LAR significantly reduced liver volume; reductions were maintained for 2 years after treatment ended. Octreotide LAR was well-tolerated. ClinicalTrials.gov number: NCT02119052.
RCT Entities:
BACKGROUND & AIMS: 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 (octreotideLAR), a somatostatin inhibitor, vs placebo in these patients. METHODS: 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 octreotideLAR (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. RESULTS: Baseline characteristics were similar between groups. After 3 years, TLV decreased by 130.2 ± 133.2 mL in patients given octreotideLAR (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 octreotideLAR 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. CONCLUSIONS: In a placebo-controlled study of patients with polycystic kidney and liver disease, 3 years of treatment with octreotideLAR significantly reduced liver volume; reductions were maintained for 2 years after treatment ended. OctreotideLAR was well-tolerated. ClinicalTrials.gov number: NCT02119052.
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