| 초록 |
Objectives: Tolvaptan is an established disease-modifying therapy for autosomal dominant polycystic kidney disease (ADPKD), supported by randomized controlled trials and prospective cohort studies. However, real-world evidence reflecting routine clinical use, dosing patterns, effectiveness, and safety remains limited in Korea. Methods: We included 400 adult patients with ADPKD who initiated tolvaptan therapy after the introduction of national health insurance coverage in Korea. Disease progression trajectories were analyzed using interrupted time-series (ITS) models for estimated glomerular filtration rate (eGFR) and height-adjusted total kidney volume (htTKV). Results: ITS analysis showed significant attenuation of the eGFR slope (+0.29 mL/min/1.73 m² per month, P < 0.001) after tolvaptan initiation. htTKV growth slopes were also significantly reduced after treatment initiation (−9.73 mL/month, P = 0.015). Dose-based analyses demonstrated greater eGFR slope attenuation in higher-dose groups. The most common tolvaptan-related adverse events were aquaretic symptoms and LFT abnormalities. No cases of severe hepatotoxicity, fulminant hepatic failure, or treatment-related mortality were observed. Conclusion: Tolvaptan therapy in Korean patients with ADPKD was associated with significant attenuation of kidney function decline and kidney volume progression, with acceptable safety and tolerability under routine clinical practice conditions. |