OBJECTIVE: To observe the effect on total liver volume (TLV) on and off therapy in selected symptomatic patients with autosomal dominant polycystic kidney disease (ADPKD) or autosomal dominant polycystic liver disease (PLD) who received octreotide long-acting release (OctLAR) for up to 4 years. PATIENTS AND METHODS: Twenty-eight of 42 participants in a prospective 2-year clinical trial ofOctLAR (40 mg monthly) consisting of double-blind, randomized (year 1) and open-label treatment (year 2) phases reenrolled in a 2-year open-label extension (OLE) study after being off OctLAR a mean of 8.3 months (original study: July 1, 2007, through June 30, 2013). Participants underwent magnetic resonance imaging at baseline, years 1 and 2, reenrollment, and study completion. Primary end point: change in TLV; secondary end points: changes in total kidney volume, glomerular filtration rate, quality of life (QoL), safety, vital signs, and laboratory parameters. RESULTS: Twenty-five participants (59.5%) completed the OLE. Off therapy, TLVs increased a mean ± SD of 3.4%±8.2% per year; after resuming therapy, TLVs decreased a mean ± SD of -4.7%±6.1% per year. Despite regrowth off treatment, overall reductions were observed, with a median (interquartile range) TLV of 4047 mL (3107-7402 mL) at baseline and 3477 (2653-7131 mL) at study completion (-13.2%; P<.001) and with improved health-related QoL. Total kidney volumes increased, and glomerular filtration rates declined from 58.2 mL/min to 54.5 mL/min (n=16) in patients with ADPKD on therapy from baseline to study completion. CONCLUSION: Therapy with OctLAR over 4 years in selected patients with symptomatic PLD arrested PLD progression, alleviating symptoms and improving health-related QoL. Discontinuation led to organ regrowth. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00426153.
RCT Entities:
OBJECTIVE: To observe the effect on total liver volume (TLV) on and off therapy in selected symptomatic patients with autosomal dominant polycystic kidney disease (ADPKD) or autosomal dominant polycystic liver disease (PLD) who received octreotide long-acting release (OctLAR) for up to 4 years. PATIENTS AND METHODS: Twenty-eight of 42 participants in a prospective 2-year clinical trial of OctLAR (40 mg monthly) consisting of double-blind, randomized (year 1) and open-label treatment (year 2) phases reenrolled in a 2-year open-label extension (OLE) study after being off OctLAR a mean of 8.3 months (original study: July 1, 2007, through June 30, 2013). Participants underwent magnetic resonance imaging at baseline, years 1 and 2, reenrollment, and study completion. Primary end point: change in TLV; secondary end points: changes in total kidney volume, glomerular filtration rate, quality of life (QoL), safety, vital signs, and laboratory parameters. RESULTS: Twenty-five participants (59.5%) completed the OLE. Off therapy, TLVs increased a mean ± SD of 3.4%±8.2% per year; after resuming therapy, TLVs decreased a mean ± SD of -4.7%±6.1% per year. Despite regrowth off treatment, overall reductions were observed, with a median (interquartile range) TLV of 4047 mL (3107-7402 mL) at baseline and 3477 (2653-7131 mL) at study completion (-13.2%; P<.001) and with improved health-related QoL. Total kidney volumes increased, and glomerular filtration rates declined from 58.2 mL/min to 54.5 mL/min (n=16) in patients with ADPKD on therapy from baseline to study completion. CONCLUSION: Therapy with OctLAR over 4 years in selected patients with symptomatic PLD arrested PLD progression, alleviating symptoms and improving health-related QoL. Discontinuation led to organ regrowth. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00426153.
Authors: Piero Ruggenenti; Andrea Remuzzi; Patrizia Ondei; Giorgio Fasolini; Luca Antiga; Bogdan Ene-Iordache; Giuseppe Remuzzi; Franklin H Epstein Journal: Kidney Int Date: 2005-07 Impact factor: 10.612
Authors: Loes van Keimpema; Frederik Nevens; Ragna Vanslembrouck; Martijn G H van Oijen; Aswin L Hoffmann; Helena M Dekker; Robert A de Man; Joost P H Drenth Journal: Gastroenterology Date: 2009-07-29 Impact factor: 22.682
Authors: Vicente E Torres; Arlene B Chapman; Olivier Devuyst; Ron T Gansevoort; Jared J Grantham; Eiji Higashihara; Ronald D Perrone; Holly B Krasa; John Ouyang; Frank S Czerwiec Journal: N Engl J Med Date: 2012-11-03 Impact factor: 91.245
Authors: Kyongtae T Bae; Fang Zhu; Arlene B Chapman; Vicente E Torres; Jared J Grantham; Lisa M Guay-Woodford; Deborah A Baumgarten; Bernard F King; Louis H Wetzel; Philip J Kenney; Marijn E Brummer; William M Bennett; Saulo Klahr; Catherine M Meyers; Xiaoling Zhang; Paul A Thompson; J Philip Miller Journal: Clin J Am Soc Nephrol Date: 2005-10-26 Impact factor: 8.237
Authors: Richard Haynes; Natalie Staplin; Jonathan Emberson; William G Herrington; Charles Tomson; Lawrence Agodoa; Vladimir Tesar; Adeera Levin; David Lewis; Christina Reith; Colin Baigent; Martin J Landray Journal: Am J Kidney Dis Date: 2014-03-05 Impact factor: 8.860
Authors: Carsten Bergmann; Lisa M Guay-Woodford; Peter C Harris; Shigeo Horie; Dorien J M Peters; Vicente E Torres Journal: Nat Rev Dis Primers Date: 2018-12-06 Impact factor: 52.329
Authors: Paula Olaizola; Pedro M Rodrigues; Francisco J Caballero-Camino; Laura Izquierdo-Sanchez; Patricia Aspichueta; Luis Bujanda; Nicholas F Larusso; Joost P H Drenth; Maria J Perugorria; Jesus M Banales Journal: Nat Rev Gastroenterol Hepatol Date: 2022-05-13 Impact factor: 73.082
Authors: William J Brock; James J Beaudoin; Jason R Slizgi; Mingming Su; Wei Jia; Sharin E Roth; Kim L R Brouwer Journal: Int J Toxicol Date: 2018 Mar/Apr Impact factor: 2.032
Authors: Anatoliy I Masyuk; Tatyana V Masyuk; Maria J Lorenzo Pisarello; Jingyi Francess Ding; Lorena Loarca; Bing Q Huang; Nicholas F LaRusso Journal: Hepatology Date: 2018-02-01 Impact factor: 17.425
Authors: Marie C Hogan; Julie A Chamberlin; Lisa E Vaughan; Angela L Waits; Carly Banks; Kathleen Leistikow; Troy Oftsie; Chuck Madsen; Marie Edwards; James Glockner; Walter K Kremers; Peter C Harris; Nicholas F LaRusso; Vicente E Torres; Tatyana V Masyuk Journal: Clin J Am Soc Nephrol Date: 2020-08-25 Impact factor: 8.237