Lanreotide Reduces Liver Growth In Patients With Autosomal Dominant Polycystic Liver and Kidney Disease

BACKGROUND AND AIMS: Polycystic liver disease is the most common extra-renal manifestation of autosomal dominant polycystic kidney disease (ADPKD). There is need for robust long-term evidence for the volume-reducing effect of somatostatin analogues. We made use of data from an open-label, randomized trial to determine the effects of lanreotide on height-adjusted liver volume (hTLV) and combined height-adjusted liver and kidney volume (hTLKV) in patients with ADPKD.

METHODS: We performed a 120-week study comparing the reno-protective effects of lanreotide vs standard care in 305 patients with ADPKD (the DIPAK-1 study). For this analysis we studied the 175 patients with polycystic liver disease, with hepatic cysts identified by magnetic resonance imaging and liver volume ≥2000 ml. Of these, 93 patients were assigned to a group that received lanreotide (120 mg subcutaneously every 4 weeks) and 82 to a group that received standard care (blood pressure coKN2ntrol, a sodium-restricted diet, and anti-hypertensive agents). The primary endpoint was percentage change in hTLV between baseline and end of treatment (week 120). A secondary endpoint was change in hTLKV.

RESULTS: At 120 weeks, hTLV decreased by 1.99% in the lanreotide group (95% CI, -4.21 to 0.24) and increased by 3.92% in the control group (95% CI, 1.56-6.28). Compared to controls, lanreotide reduced the growth of hTLV by 5.91% (95% CI, -9.18 to -2.63; P<.001). Growth of hTLV was still reduced by 3.87% at 4 months after the last injection of lanreotide compared to baseline (95% CI, -7.55 to -0.18; P=.04). Lanreotide reduced growth of hTLKV by 7.18% compared with controls (95% CI, -10.25 to -4.12; P<.001).

CONCLUSION: In this subanalysis of a randomized trial of patients with polycystic liver disease due to ADPKD, lanreotide for 120 weeks reduced the growth of liver and combined liver- and kidney volume. This effect was still present 4 months after the last injection of lanreotide. ClinicalTrials.gov no: NCT01616927.