Pansomatostatin Agonist Pasireotide Long-Acting Release for Patients with Autosomal Dominant Polycystic Kidney or Liver Disease with Severe Liver Involvement: A Randomized Clinical Trial

• Published in: Clinical journal of the American Society of Nephrology 2020-09, Vol.15 (9), p.1267-1278
• Main Authors: Hogan, Marie C, Chamberlin, Julie A, Vaughan, Lisa E, Waits, Angela L, Banks, Carly, Leistikow, Kathleen, Oftsie, Troy, Madsen, Chuck, Edwards, Marie, Glockner, James, Kremers, Walter K, Harris, Peter C, LaRusso, Nicholas F, Torres, Vicente E, Masyuk, Tatyana V
• Format: Article
• Language: English
• Subjects: Adult; Cysts - drug therapy; Cysts - pathology; Disease Progression; Double-Blind Method; Female; Glomerular Filtration Rate - drug effects; Humans; Kidney - drug effects; Kidney - pathology; Kidney - physiopathology; Liver - drug effects; Liver - pathology; Liver Diseases - drug therapy; Liver Diseases - pathology; Male; Middle Aged; Minnesota; Organ Size; Original; Polycystic Kidney, Autosomal Dominant - drug therapy; Polycystic Kidney, Autosomal Dominant - pathology; Polycystic Kidney, Autosomal Dominant - physiopathology; Quality of Life; Severity of Illness Index; Somatostatin - adverse effects; Somatostatin - analogs & derivatives; Somatostatin - therapeutic use; Time Factors; Treatment Outcome
• ISSN: 1555-9041; 1555-905X; 1555-905X
• DOI: 10.2215/CJN.13661119

We assessed safety and efficacy of another somatostatin receptor analog, pasireotide long-acting release, in severe polycystic liver disease and autosomal dominant polycystic kidney disease. Pasireotide long-acting release, with its broader binding profile and higher affinity to known somatostatin receptors, has potential for greater efficacy. Individuals with severe polycystic liver disease were assigned in a 2:1 ratio in a 1-year, double-blind, randomized trial to receive pasireotide long-acting release or placebo. Primary outcome was change in total liver volume; secondary outcomes were change in total kidney volume, eGFR, and quality of life. Of 48 subjects randomized, 41 completed total liver volume measurements ( =29 pasireotide long-acting release and =12 placebo). From baseline, there were -99±189 ml/m absolute and -3%±7% change in annualized change in height-adjusted total liver volume (from 2582±1381 to 2479±1317 ml/m) in the pasireotide long-acting release group compared with 136±117 ml/m absolute and 6%±7% increase (from 2387±759 to 2533±770 ml/m) in placebo (