Treatment for patients with autosomal dominant polycystic kidney disease in the chronic kidney disease without kidney replacement therapy in real-world clinical practice: a descriptive retrospective cohort study

Abstract

Competing Interests: Kayoko Mizuno has been employed by the Department of Digital Health and Epidemiology with support from Eisai Co., Ltd. and Kyowa Kirin Co., Ltd.; and has received grants from Japan Society for the Promotion of Science (JSPS). Motoko Yanagita received research grants from Mitsubishi Tanabe Pharma and Boehringer Ingelheim. Koji Kawakami has received research funds from Eisai Co., Ltd., Kyowa Kirin Co., Ltd., Mitsubishi Corporation, OMRON Corporation, Real World Data Co., Ltd., Sumitomo Pharma Co., Ltd., and Toppan Inc.; consulting fees from Advanced Medical Care Inc., JMDC Inc., LEBER Inc., and Shin Nippon Biomedical Laboratories Ltd.; executive compensation from Cancer Intelligence Care Systems, Inc.; honoraria from Chugai Pharmaceutical Co., Ltd., Kaken Pharmaceutical Co., Ltd., Mitsubishi Chemical Holdings Corporation, Mitsubishi Corporation, Pharma Business Academy, and Toppan Inc.; and held stock in Real World Data Co., Ltd. Kazunori Sakoda, Kayoko Mizuno, Tomotsugu Seki, Kanna Shinkawa, Yuriko Ohara, Ayano Hayashi, Satomi Yoshida, and Masato Takeuchi have no conflict of interest to declare.; Background: In real-world clinical practice, treatments selected for patients with autosomal dominant polycystic kidney disease (ADPKD) in the chronic kidney disease (CKD) without kidney replacement therapy (KRT) have not been reported. This study investigated the oral treatments used in these patients and the changes in their use in recent years. Additionally, we studied the factors affecting tolvaptan dose reduction or discontinuation.; Methods: This retrospective cohort study was conducted using the medical records of 160 hospitals in Japan. Patients with ADPKD or polycystic kidney disease registered on the database between January 2014 and December 2020 were selected. Changes in prescription proportions over time were assessed using the Cochran-Armitage test. We focused on patients prescribed with >15 mg of tolvaptan daily to identify the factors related to its dose reduction or discontinuation and used Multivariate Cox regression analysis to evaluate them.; Results: Tolvaptan use in patients with ADPKD in the CKD without KRT stage has increased. As of 2020, 25% of patients were treated with tolvaptan. Overall, 3639 patients with ADPKD were enrolled in the database, of whom 156 were treated with tolvaptan. Of these, 64 patients (41%) reduced or discontinued tolvaptan during the observation period. The presence of an estimated glomerul…