title: Young women with polycystic liver disease respond best to somatostatin analogues: a pooled analysis of individual patient data.
abstract: OBJECTIVE
  Clinical trials have shown that in patients with polycystic liver disease (PLD), short-term treatment with somatostatin analogues (SAs) reduces liver volumes by 4.5%-5.9%, compared with placebo. However, the effects of SA therapy vary among individuals. We collected data from individual patients with PLD to identify subgroups that benefit most from SA therapy.
  
  
  METHODS
  We analyzed data from 107 patients with PLD from 3 randomized placebo-controlled trials (67 received SAs, 52 received placebo). We used multiple linear regression analysis to determine the effects of SAs based on patients' age, sex, baseline liver volume, and diagnosis (autosomal dominant polycystic liver or kidney disease). The primary outcome was change in liver volume after 6-12 months of treatment.
  
  
  RESULTS
  The effects of SA therapy did not differ significantly among patients with different diagnoses or baseline liver volumes; the overall difference in liver volume between groups receiving SAs therapy vs placebo was 5.3% (P < .001). Among subjects given placebo, young women (48 years old or younger) had the greatest increase in polycystic liver volume (4.8%; 95% confidence interval: 2.2%-7.4%), and mean liver volumes did not increase in older women and men. Women 48 years old or younger had a greater response to therapy (a reduction in liver volume of 8.0% compared with placebo; P < .001) than older women (a reduction in liver volume of 4.1% compared with placebo; P = .022).
  
  
  CONCLUSIONS
  Based on a pooled analysis of data from individual patients with PLD, treatment with somatostatin analogues is equally effective for patients with autosomal dominant polycystic kidney disease or polycystic liver disease; efficacy does not depend on size of the polycystic liver. Young female patients appear to have the greatest benefit from 6-12 months of SA therapy, which might avert the progressive course of the disease in this specific group.
authors.affiliation: Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, The Netherlands. t.gevers@mdl.umcn.nl
authors.forename: Tom J G
authors.initials: TJ
authors.lastname: Gevers
authors.forename: Joanna
authors.initials: J
authors.lastname: Inthout
authors.forename: Anna
authors.initials: A
authors.lastname: Caroli
authors.forename: Piero
authors.initials: P
authors.lastname: Ruggenenti
authors.forename: Marie C
authors.initials: MC
authors.lastname: Hogan
authors.forename: Vicente E
authors.initials: VE
authors.lastname: Torres
authors.forename: Frederik
authors.initials: F
authors.lastname: Nevens
authors.forename: Joost P H
authors.initials: JP
authors.lastname: Drenth
mesh_terms: D000328:Adult; D000368:Aged; D003560:Cysts / Q000188:drug therapy* / Q000473:pathology; D005260:Female; D006801:Humans; D016014:Linear Models; D008099:Liver / Q000473:pathology*; D008107:Liver Diseases / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D015282:Octreotide / Q000627:therapeutic use; D009929:Organ Size; D010456:Peptides, Cyclic / Q000627:therapeutic use; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy*; D016032:Randomized Controlled Trials as Topic; D013004:Somatostatin / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D010456:Peptides, Cyclic; C060347:lanreotide; D013004:Somatostatin; C517782:pasireotide; D015282:Octreotide
keywords: ADPKD; CI; CT; Drug Therapy; IPD; Liver Cysts; PCLD; PLD; RCT; SA; autosomal dominant polycystic kidney disease; autosomal dominant polycystic liver disease; computed tomography; confidence interval; individual patient data; polycystic liver disease; randomized controlled trial; somatostatin analogue