The long-term outcome of patients with polycystic liver disease treated with lanreotide

• Published in: Alimentary pharmacology & therapeutics Vol. 35; no. 2; pp. 266 - 274
• Main Authors: Chrispijn, M., Nevens, F., Gevers, T. J. G., Vanslembrouck, R., van Oijen, M. G. H., Coudyzer, W., Hoffmann, A. L., Dekker, H. M., de Man, R. A., van Keimpema, L., Drenth, J. P. H.
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.01.2012; Blackwell
• Subjects: Adult; Antineoplastic Agents - therapeutic use; Biological and medical sciences; Cysts - drug therapy; Cysts - physiopathology; Digestive system; Female; Gastroenterology. Liver. Pancreas. Abdomen; Humans; Liver - drug effects; Liver Diseases - drug therapy; Liver Diseases - physiopathology; Liver Function Tests; Liver. Biliary tract. Portal circulation. Exocrine pancreas; Male; Medical sciences; Middle Aged; Peptides, Cyclic - therapeutic use; Pharmacology. Drug treatments; Somatostatin - analogs & derivatives; Somatostatin - therapeutic use; Statistics as Topic; Time Factors; Treatment Outcome; Tumors; Human; Prognosis; Peptides; Polycystic liver; Hepatic disease; Long term; Genetic disease; Octapeptide; Treatment; Lanreotide; Cyst; Digestive diseases; Benign neoplasm
• ISSN: 0269-2813; 1365-2036
• DOI: 10.1111/j.1365-2036.2011.04923.x

Aliment Pharmacol Ther 2012; 35: 266–274 Summary Background  Polycystic liver disease (PLD) is a phenotypical expression of autosomal dominant polycystic kidney disease and isolated polycystic liver disease. Somatostatin analogues, such as lanreotide, reduce polycystic liver volume. Aim  To establish long‐term outcome and safety of lanreotide. Methods  This was an open‐label, observational extension study of a 6‐month, randomised, placebo‐controlled trial with lanreotide (120 mg/month) in PLD. The length of total treatment was 12 months. Primary endpoint was relative change in liver volume, as determined by CT‐volumetry after 12 months of treatment. We offered patients a CT scan 6 months after stopping lanreotide. Results  A total of 41/54 (76%) patients participated in the extension study. Liver volume decreased by 4% (IQR −8% to −1%) after 12 months of treatment. The greatest effect was observed during the first 6 months of treatment (decrease of 4% (IQR −6% to −1%)). Liver volume remained unchanged during the following 6 months. We found that liver volume increased by 4% (IQR 0–6%) 6 months after end of treatment (n = 22). Conclusions  Lanreotide reduces liver volume within the first 6 months of treatment and the beneficial effect is maintained in the following 6 months. Stopping results in recurrence of polycystic liver growth. This suggests that continuous use of lanreotide is needed to maintain its effect.