Effect of lanreotide on polycystic liver and kidneys in autosomal dominant polycystic kidney disease: an observational trial.

BACKGROUND & AIM: Several trials have demonstrated that somatostatin analogues decrease liver volume in mixed populations of patients with autosomal dominant polycystic kidney disease (ADPKD) and isolated polycystic liver disease. Chronic renal dysfunction in ADPKD may affect treatment efficacy of lanreotide and possibly enhances risk for adverse events. The aim of this open-label clinical trial (RESOLVE trial) was to assess the efficacy of 6-month lanreotide treatment, 120 mg, subcutaneously every 4 weeks in ADPKD patients with symptomatic polycystic liver disease.

METHODS

Primary outcome was change in liver volume after 6 months; secondary outcomes were changes in kidney volume, estimated glomerular filtration rate (eGFR), symptom relief and health-related quality of life (Euro-Qol5D). We excluded patients with an eGFR <30 ml/min/1.73 m(2) . We used the Wilcoxon signed-rank test or paired two-sided t-test to analyze within-group differences.

RESULTS

We included 43 ADPKD patients with polycystic liver disease (84% female, median age 50 years, mean eGFR 63 ml/min/1.73 m(2) ). Median liver volume decreased from 4859 ml to 4595 ml (-3.1%; P < 0.001), and median kidney volume decreased from 1023 ml to 1012 ml (-1.7%; P = 0.006). eGFR declined 3.5% after the first injection, remained stable up to study end, to decline again after lanreotide withdrawal. Lanreotide significantly relieved post-prandial fullness, shortness of breath and abdominal distension. Three participants had a suspected episode of hepatic or renal cyst infection during this study.

CONCLUSION

Lanreotide reduced polycystic liver and kidney volumes and decreases symptoms in ADPKD patients. Moreover, eGFR decreased acutely after starting lanreotide, stabilized thereafter and declined again after withdrawal.

TRIAL REGISTRATION NUMBER: Clinical trials.gov NCT01354405 (REGISTRATION: 13 May 2011).