BOSTON, MA—The investigative agent, pasireotide LAR (SOM230) was found to delay the progressive increase in liver volume and total kidney volume in patients with polycystic liver disease (PLD) and autosomal dominant polycystic kidney disease (ADPKD), in a study presented at The Liver Meeting® 2016.

Octreotide LAR, a somatostatin receptor analog, has been reported to reduce liver volume, improve quality of life in symptomatic PLD and slow glomerular filtration rate (GFR) decline in ADPKD. Maria V. Irazabal, MD, and colleagues from the Mayo Clinic, Rochester, MN, aimed to assess the safety and efficacy of pasireotide LAR in severe PLD and ADPKD. Pasireotide, a novel multi-receptor ligand somatostatin analog, possesses a “broader binding profile and higher affinity to known somatostatin receptors with potential for greater efficacy.” 

In the 1-year, double-blind randomized trial, 48 study patients were assigned 2:1 to receive pasireotide LAR 60mg or placebo every 28 days; patients were stratified by ADPKD and ADPLD.

“Primary endpoint was change in liver volume; secondary endpoints were change in kidney volume, estimated GFR [eGFR], and quality of life [QOL],” explained Dr. Irazabal. Forty patients completed liver volume measurements at 12 months. 

The researchers reported a 3.3% decrease in annualized change in liver volume (4271 ± 2373mL to 4104 ± 2265mL) in the pasireotide LAR group vs. a 6.3% increase in liver volume (4047 ± 1298mL to 4294 ± 1314mL) in the placebo group (P=0.001). 

Regarding kidney volume, researchers reported a 1.1% decrease in the pasireotide LAR group vs. a 3.9% increase in the placebo group (P=0.024). 

“Changes in eGFR were not different between groups,” noted Dr. Irazabal. 

Pasireotide LAR the target dose, was reached in 87.5% of patients within the first year. Patients in the pasireotide LAR group, however, experienced a higher incidence of adverse events such as hyperglycemia (73% vs. 20%; P=0.003) and diabetes (30% vs. 0%; P=0.042) compared to the placebo group. 

Overall, pasireotide LAR slowed the progressive growth in both liver and total kidney volume in patients with PLD and ADPKD. Bigger studies are warranted to determine the impact of somatostatin receptor analogs on eGFR decline, concluded study authors.