Department of Nephrology, Radboud University Medical Center, Nijmegen, Netherlands.
Department of Nephrology and Medical Intensive Care, Charité Universitätsmedizin Berlin, Berlin, Germany.
Transpl Int. 2022 May 20;35:10139. doi: 10.3389/ti.2022.10139. eCollection 2022.
Clinical study endpoints that assess the efficacy of interventions in patients with chronic renal insufficiency can be adopted for use in kidney transplantation trials, given the pathophysiological similarities between both conditions. Kidney dysfunction is reflected in the glomerular filtration rate (GFR), and although a predefined (e.g., 50%) reduction in GFR was recommended as an endpoint by the European Medicines Agency (EMA) in 2016, many other endpoints are also included in clinical trials. End-stage renal disease is strongly associated with a change in estimated (e)GFR, and eGFR trajectories or slopes are increasingly used as endpoints in clinical intervention trials in chronic kidney disease (CKD). Similar approaches could be considered for clinical trials in kidney transplantation, although several factors should be taken into account. The present Consensus Report was developed from documentation produced by the European Society for Organ Transplantation (ESOT) as part of a Broad Scientific Advice request that ESOT submitted to the EMA in 2020. This paper provides a contemporary discussion of primary endpoints used in clinical trials involving CKD, including proteinuria and albuminuria, and evaluates the validity of these concepts as endpoints for clinical trials in kidney transplantation.
临床研究终点可用于评估慢性肾功能不全患者干预措施的疗效，因为这两种疾病在病理生理学上有相似之处。肾功能障碍反映在肾小球滤过率（GFR）中，尽管欧洲药品管理局（EMA）在 2016 年建议将 GFR 降低（例如 50%）作为终点，但许多其他终点也包含在临床试验中。终末期肾病与估计肾小球滤过率（eGFR）的变化密切相关，eGFR 轨迹或斜率越来越多地被用作慢性肾脏病（CKD）临床干预试验的终点。类似的方法可以考虑用于肾移植临床试验，但应考虑几个因素。本共识报告是根据欧洲器官移植学会（ESOT）在 2020 年向 EMA 提交的广泛科学咨询请求中产生的文件编写的。本文对涉及 CKD 的临床试验中使用的主要终点（包括蛋白尿和白蛋白尿）进行了当代讨论，并评估了这些概念作为肾移植临床试验终点的有效性。
