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Abstract：
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 glomerular filtration rate <60 mL/min/1.73 m2 at the beginning of the treatment was associated with a higher risk of tolvaptan dose reduction or discontinuation.
CONCLUSIONS: The proportion of patients with ADPKD treated with high-dose tolvaptan is increasing. However, patients with late-stage CKD tended to reduce or discontinue tolvaptan.
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 glomerular filtration rate <60 mL/min/1.73 m2 at the beginning of the treatment was associated with a higher risk of tolvaptan dose reduction or discontinuation.
CONCLUSIONS: The proportion of patients with ADPKD treated with high-dose tolvaptan is increasing. However, patients with late-stage CKD tended to reduce or discontinue tolvaptan.
摘要：
背景：在现实世界的临床实践中，在慢性肾脏病(CKD)中常染色体显性遗传性多囊肾病(ADPKD)患者中选择的无肾脏替代治疗(KRT)的治疗方法尚未报道.这项研究调查了近年来这些患者使用的口服治疗方法及其使用的变化。此外,我们研究了影响托伐普坦减量或停药的因素.
方法：本回顾性队列研究使用日本160家医院的医疗记录进行。选择2014年1月至2020年12月在数据库中注册的ADPKD或多囊肾病患者。使用Cochran-Armitage测试评估处方比例随时间的变化。我们关注每天服用>15mg托伐普坦的患者，以确定与剂量减少或停药相关的因素，并使用多因素Cox回归分析来评估这些因素。
结果：托伐普坦在无KRT阶段的CKD患者中的使用有所增加。截至2020年，25%的患者接受托伐普坦治疗。总的来说,3639例ADPKD患者纳入数据库，其中156人接受托伐普坦治疗。其中,64例患者（41%）在观察期间减少或停用托伐普坦。在治疗开始时估计的肾小球滤过率<60mL/min/1.73m2的存在与托伐普坦剂量减少或停药的较高风险相关。
结论：使用大剂量托伐普坦治疗的ADPKD患者比例正在增加。然而,晚期CKD患者倾向于减少或停用托伐普坦.
方法：本回顾性队列研究使用日本160家医院的医疗记录进行。选择2014年1月至2020年12月在数据库中注册的ADPKD或多囊肾病患者。使用Cochran-Armitage测试评估处方比例随时间的变化。我们关注每天服用>15mg托伐普坦的患者，以确定与剂量减少或停药相关的因素，并使用多因素Cox回归分析来评估这些因素。
结果：托伐普坦在无KRT阶段的CKD患者中的使用有所增加。截至2020年，25%的患者接受托伐普坦治疗。总的来说,3639例ADPKD患者纳入数据库，其中156人接受托伐普坦治疗。其中,64例患者（41%）在观察期间减少或停用托伐普坦。在治疗开始时估计的肾小球滤过率<60mL/min/1.73m2的存在与托伐普坦剂量减少或停药的较高风险相关。
结论：使用大剂量托伐普坦治疗的ADPKD患者比例正在增加。然而,晚期CKD患者倾向于减少或停用托伐普坦.
