With limited real-world data on the safety and efficacy of tolvaptan since its approval to treat ADPKD in 2014, investigators conducted post-marketing surveillance to evaluate the long-term safety and efficacy of the agent in patients with ADPKD in real-world clinical settings with a mean age of 49.7, and baseline total kidney volume and eGFR levels of 2,158 mL and 44.4 mL/min/1.73m2, respectively. Among those with chronic kidney disease, 27% had stage G3b and 30% has stage G4. The most frequently reported adverse drug reactions were abnormal hepatic function (8.3%), thirst (8.2%), and hyperuricaemia (6.9%).
Annual rates of percentage change in total kidney volume (TKV) were reduced from 11.68% per year prior to tolvaptan treatment to 2.74% per year following treatment. Annual change in eGFR was reduced from -3.31 mL/min/1.73m2 prior to treatment to -2.28 mL/min/1.73m2 following tolvaptan treatment. “There were no major problems with safety of tolvaptan treatment and comparable efficacy for TKV and eGFR was observed in relation to the previous pivotal two randomized control trials in this post-marketing surveillance,” wrote the study authors.