Department II of Internal Medicine and Center for Molecular Medicine, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany.
CECAD, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany.
Nephrol Dial Transplant. 2022 Apr 25;37(5):825-839. doi: 10.1093/ndt/gfab312.
Approval of the vasopressin V2 receptor antagonist tolvaptan-based on the landmark TEMPO 3:4 trial-marked a transformation in the management of autosomal dominant polycystic kidney disease (ADPKD). This development has advanced patient care in ADPKD from general measures to prevent progression of chronic kidney disease to targeting disease-specific mechanisms. However, considering the long-term nature of this treatment, as well as potential side effects, evidence-based approaches to initiate treatment only in patients with rapidly progressing disease are crucial. In 2016, the position statement issued by the European Renal Association (ERA) was the first society-based recommendation on the use of tolvaptan and has served as a widely used decision-making tool for nephrologists. Since then, considerable practical experience regarding the use of tolvaptan in ADPKD has accumulated. More importantly, additional data from REPRISE, a second randomized clinical trial (RCT) examining the use of tolvaptan in later-stage disease, have added important evidence to the field, as have post hoc studies of these RCTs. To incorporate this new knowledge, we provide an updated algorithm to guide patient selection for treatment with tolvaptan and add practical advice for its use.
托伐普坦（vasopressin V2 receptor antagonist）基于具有里程碑意义的 TEMPO 3:4 试验获得批准，这标志着常染色体显性多囊肾病（autosomal dominant polycystic kidney disease，ADPKD）的治疗发生了转变。这一进展将 ADPKD 的患者护理从预防慢性肾脏病进展的一般措施推进到针对特定疾病机制的治疗。然而，鉴于这种治疗的长期性以及潜在的副作用，仅在疾病快速进展的患者中启动治疗的基于证据的方法至关重要。2016 年，欧洲肾脏协会（European Renal Association，ERA）发布的立场声明是首个关于托伐普坦使用的基于学会的建议，它已成为肾病学家广泛使用的决策工具。此后，在 ADPKD 中使用托伐普坦积累了相当多的实际经验。更重要的是，第二项评估托伐普坦在晚期疾病中应用的随机临床试验（randomized clinical trial，RCT）REPRISE 的附加数据，以及这些 RCT 的事后研究，为该领域提供了重要的证据。为了纳入这些新知识，我们提供了一个更新的算法来指导托伐普坦治疗的患者选择，并添加了其使用的实用建议。
