Tolvaptan in autosomal dominant polycystic kidney disease — a real-life experience

Introduction: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic disease of the kidney, leading to end-stage kidney disease (ESKD) in a large proportion of affected individuals. The only approved therapy to slow the progression of chronic kidney disease (CKD) secondary to ADPKD is tolvaptan. The following analysis aimed to present the experience of the centre with tolvaptan used for ADPKD. Materials and methods: Retrospective analysis of single centre data. The study group consisted of 13 patients who started treatment with tolvaptan. The control group consisted of 13 patients who refused to be treated with tolvaptan. Results: In the study group, 2 patients (15%) discontinued tolvaptan due to the side effects. The intention to treat (ITT) analysis showed that among both groups progression of CKD occurred during the observation period. No statistically significant difference in the median change of estimated glomerular filtration rate (eGFR) was noticed between the study and the control group. Moreover, no statistically significant difference in the median change of eGFR was noticed between the pre-study period and the observation period both in the study group and in the control group. Conclusions: According to the following results, tolvaptan is not effective in slowing the progression of CKD in patients with ADPKD in real-life settings. Further observations are needed to confirm these results.