title: Efficacy of 4 Years of Octreotide Long-Acting Release Therapy in Patients With Severe Polycystic Liver Disease.
abstract: 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.
  
  
  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.
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  clinicaltrials.gov Identifier: NCT00426153.
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN. Electronic address: hogan.marie@mayo.edu.
authors.forename: Marie C
authors.initials: MC
authors.lastname: Hogan
authors.affiliation: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN.
authors.forename: Tetyana
authors.initials: T
authors.lastname: Masyuk
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, MN.
authors.forename: Eric
authors.initials: E
authors.lastname: Bergstralh
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, MN.
authors.forename: Bill
authors.initials: B
authors.lastname: Li
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, MN.
authors.forename: Walter K
authors.initials: WK
authors.lastname: Kremers
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, MN.
authors.forename: Lisa E
authors.initials: LE
authors.lastname: Vaughan
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: Angela
authors.initials: A
authors.lastname: Ihrke
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: Amanda L
authors.initials: AL
authors.lastname: Severson
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: Maria V
authors.initials: MV
authors.lastname: Irazabal
authors.affiliation: Department of Radiology, Mayo Clinic, Rochester, MN.
authors.forename: James
authors.initials: J
authors.lastname: Glockner
authors.affiliation: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN.
authors.forename: Nicholas F
authors.initials: NF
authors.lastname: LaRusso
authors.affiliation: Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: Vicente E
authors.initials: VE
authors.lastname: Torres
mesh_terms: D018931:Antineoplastic Agents, Hormonal / Q000627:therapeutic use*; D003560:Cysts / Q000188:drug therapy* / Q000473:pathology; D003692:Delayed-Action Preparations; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D008107:Liver Diseases / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D015282:Octreotide / Q000627:therapeutic use*; D009929:Organ Size; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000473:pathology; D011446:Prospective Studies; D016896:Treatment Outcome
chemical_list: D018931:Antineoplastic Agents, Hormonal; D003692:Delayed-Action Preparations; D015282:Octreotide