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Reducing polycystic liver volume in ADPKD: effects of somatostatin analogue octreotide
Anna Caroli1, Luca Antiga, Mariateresa Cafaro
1Biomedical Engineering, Mario Negri Institute for Pharmacological Research, Via Gavazzeni, 11, 24125 Bergamo, Italy.
Background And Objectives:
No medical treatment is available for polycystic liver disease, a frequent manifestation of autosomal-dominant polycystic kidney disease (ADPKD). In a prospective, randomized, double-blind, crossover study, 6 months of octreotide (40 mg every 28 days) therapy limited kidney volume growth more effectively than placebo in 12 patients with ADPKD.
Design, Setting, Participants, & Measurements:
In this secondary, post hoc analysis of the above study, octreotide-induced changes in liver volumes compared with placebo and the relationship between concomitant changes in liver and kidney volumes were evaluated. Those analyzing liver and kidney volumes were blinded to treatment.
Results:
Liver volumes significantly decreased from 1595 +/- 478 ml to 1524 +/- 453 ml with octreotide whereas they did not appreciably change with placebo. Changes in liver volumes were significantly different between the two treatment periods (-71 +/- 57 ml versus +14 +/- 85 ml). Octreotide-induced liver volume reduction was fully explained by a reduction in parenchyma volume from 1506 +/- 431 ml to 1432 +/- 403 ml. Changes in liver volumes were significantly correlated with concomitant changes in kidney volumes (r = 0.67) during octreotide but not during placebo treatment. Liver and kidney volume changes significantly differed with both treatments (octreotide: -71 +/- 57 ml versus +71 +/- 107; placebo: +14 +/- 85 ml versus +162 +/- 114), but net reductions in liver (-85 +/- 103 ml) and kidney (-91 +/- 125 ml) volume growth on octreotide versus placebo were similar.
Conclusions:
Octreotide therapy reduces liver volumes in patients with ADPKD and is safe.
Insights
Octreotide therapy significantly reduced liver volumes in patients with autosomal-dominant polycystic kidney disease (ADPKD). This treatment also showed a correlation between liver and kidney volume changes, indicating a potential therapeutic benefit.
Area of Science:
- Nephrology
- Hepatology
- Pharmacology
Background:
- Polycystic liver disease is a common complication of autosomal-dominant polycystic kidney disease (ADPKD).
- Currently, no specific medical treatments exist for polycystic liver disease.
- Previous studies indicated octreotide's potential in managing ADPKD complications.
Purpose of the Study:
- To evaluate the effect of octreotide on liver volumes in patients with ADPKD.
- To assess the relationship between changes in liver and kidney volumes during octreotide treatment.
- To determine the safety and efficacy of octreotide in reducing liver volume in ADPKD patients.
Main Methods:
- A secondary, post hoc analysis of a prospective, randomized, double-blind, crossover study.
- 12 patients with ADPKD received 6 months of octreotide (40 mg every 28 days) or placebo.
- Liver and kidney volumes were measured and analyzed by blinded personnel.
Main Results:
- Octreotide treatment led to a significant reduction in liver volumes (1595 to 1524 ml) compared to placebo.
- The reduction in liver volume was primarily due to decreased parenchyma volume.
- Changes in liver volume correlated significantly with kidney volume changes during octreotide therapy but not placebo.
Conclusions:
- Octreotide therapy effectively reduces liver volumes in patients diagnosed with ADPKD.
- The observed reduction in liver volume suggests a potential therapeutic avenue for managing polycystic liver disease.
- Octreotide demonstrated a favorable safety profile in this patient cohort.

