Lanreotide Reduces the Volume of Polycystic Liver: A Randomized, Double-Blind, Placebo-Controlled Trial
Received 19 April 2009; accepted 10 July 2009. published online 30 July 2009.
Background & aims
Therapy for polycystic liver is invasive, expensive, and has disappointing long-term results. Treatment with somatostatin analogues slowed kidney growth in patients with polycystic kidney disease (PKD) and reduced liver and kidney volume in a PKD rodent model. We evaluated the effects of lanreotide, a somatostatin analogue, in patients with polycystic liver because of autosomal-dominant (AD) PKD or autosomal-dominant polycystic liver disease (PCLD).
Methods
We performed a randomized, double-blind, placebo-controlled trial in 2 tertiary referral centers. Patients with polycystic liver (n = 54) were randomly assigned to groups given lanreotide (120 mg) or placebo, administered every 28 days for 24 weeks. The primary end point was the difference in total liver volume, measured by computerized tomography at weeks 0 and 24. Analyses were performed on an intention-to-treat basis.
Results
Baseline characteristics were comparable for both groups, except that more patients with ADPKD were assigned to the placebo group (P = .03). The mean liver volume decreased 2.9%, from 4606 mL (95% confidence interval (CI): 547–8665) to 4471 mL (95% CI: 542–8401 mL), in patients given lanreotide. In the placebo group, the mean liver volume increased 1.6%, from 4689 mL (95% CI: 613–8765 mL) to 4895 mL (95% CI: 739–9053 mL) (P < .01). Post hoc stratification for patients with ADPKD or PCLD revealed similar changes in liver volume, with statistically significant differences in patients given lanreotide (P < .01 for both diseases).
Conclusions
In patients with polycystic liver, 6 months of treatment with lanreotide reduces liver volume.
⁎Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
‡Department of Hepatology, University Hospital Leuven, Leuven
§Department of Radiology, University Hospital Leuven, Leuven, Belgium
∥Department of Radiation Oncology, Radboud University Nijmegen Medical Centre, Nijmegen
¶Department of Radiology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
#Department of Gastroenterology and Hepatology, Erasmus Medical Centre, Rotterdam, The Netherlands
Reprint requests Address requests for reprints to: Joost P. H. Drenth, MD, PhD, Professor of Molecular Gastroenterology and Hepatology, Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, P. O. Box 9101, code 455, 6500 HB Nijmegen, The Netherlands. fax: (31) 24 354 0103
Conflicts of interest The authors disclose the following: Robert A. de Man: consulting and lecture fees and grant support from Gilead Sciences, consulting fees and grant support from Bristol–Myers Squibb, consulting fees from Novartis, and lecture fees from UCB. Joost P. H. Drenth: grant/research support from Roche. The remaining authors disclose no conflicts.
Funding Supported by Ipsen, Boulogne Billancourt, France, and a ZON-MW VIDI research grant (J.P.H.D.).
ABSTRACT
Lanreotide Reduces the Volume of Polycystic Liver: A Randomized, Double-Blind, Placebo-Controlled Trial