Lanreotide, a promising drug for treatment of polycystic liver disease

DELHI: Lanreotide shows promising results in the treatment of patients with a rare liver disease polycystic liver disease (PLD) associated with autosomal dominant polycystic kidney disease (ADPKD) in a 120-week randomized clinical trial.

The study was presented at the European Association for the Study of the Liver (EASL) annual meeting. The researchers on treating patients having PKD and polycystic liver disease found that lanreotide resulted in a reduction in liver volume compared to standard of care. According to findings, height-adjusted liver volume declined 1.99% in the lanreotide group compared to an increase of 3.92% with standard of care, for a difference of -5.91%.

Lanreotide has been shown in previous PLD studies to reduce liver volume, however, there are no robust data on the long-term volume-reducing effects of this or other somatostatin analogs. René van Aerts, MD, of Radboud University Medical Center in Nijmegen, the Netherlands, and colleagues conducted the study to evaluate the long-term efficacy of the somatostatin analog, lanreotide, in PLD associated with ADPKD. Polycystic liver disease is the most common extra-renal manifestation of this rare genetic condition and is characterized by the development of fluid-filled liver cysts that cause progressive and symptomatic liver enlargement.

The DIPAK-1 trial was a randomized 120-week open-label trial designed to examine the renoprotective effect of the treatment in patients with ADPKD. van Aerts' group examined a subset of patients who also had polycystic liver disease (defined as hepatic cysts on MRI and a liver volume ≥2,000 mL).

Of these participants, 175 met criteria for polycystic liver disease, 93 of which were randomized to lanreotide and 82 to the standard of care. The primary endpoint was the percentage change in height-adjusted liver volume between baseline and end of treatment.

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• Height-adjusted liver volume declined 1.99% in the lanreotide group compared to an increase of 3.92% with standard of care, for a difference of -5.91%.


• The reduction in the primary outcome was "not very strong," but the control group showed an increase.


• There were improvements in the secondary endpoint, a change in height-adjusted liver and kidney volume for the lanreotide group versus standard of care.


• There was still a significant difference in height-adjusted liver volume in the two groups even 4 months after the end of treatment.

"Treatment with somatostatin analogs should be considered in patients with enlarged liver and/or kidneys to prevent further disease progression," concluded the authors.