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 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.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 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.

Long-term Effects of Octreotide on Liver Volume in Patients With Polycystic Kidney and Liver Disease / Pisani, A.; Sabbatini, M.; Imbriaco, M.; Riccio, E.; Rubis, N.; Prinster, A.; Perna, A.; Liuzzi, R.; Spinelli, L.; Santangelo, M.; Remuzzi, G.; Ruggenenti, P.. - In: CLINICAL GASTROENTEROLOGY AND HEPATOLOGY. - ISSN 1542-3565. - 14:7(2016), pp. 1022-1030.e4. [10.1016/j.cgh.2015.12.049]

Long-term Effects of Octreotide on Liver Volume in Patients With Polycystic Kidney and Liver Disease

Pisani A.;Imbriaco M.;Prinster A.;Santangelo M.;
2016

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 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.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 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.
2016
Long-term Effects of Octreotide on Liver Volume in Patients With Polycystic Kidney and Liver Disease / Pisani, A.; Sabbatini, M.; Imbriaco, M.; Riccio, E.; Rubis, N.; Prinster, A.; Perna, A.; Liuzzi, R.; Spinelli, L.; Santangelo, M.; Remuzzi, G.; Ruggenenti, P.. - In: CLINICAL GASTROENTEROLOGY AND HEPATOLOGY. - ISSN 1542-3565. - 14:7(2016), pp. 1022-1030.e4. [10.1016/j.cgh.2015.12.049]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/757879
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