Nephrology and Kidney Transplantation Department, Centro de Referencia en Enfermedad Glomerular Compleja del Sistema Nacional de Salud (CSUR), Hospital Clínic, University of Barcelona, Barcelona, Spain,
Nephrology and Kidney Transplantation Department, Centro de Referencia en Enfermedad Glomerular Compleja del Sistema Nacional de Salud (CSUR), Hospital Clínic, University of Barcelona, Barcelona, Spain.
Nephron. 2023;147(3-4):152-157. doi: 10.1159/000526368. Epub 2022 Sep 9.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary cause of end-stage kidney disease. Currently, tolvaptan is the only treatment that has proven to delay disease progression. The most notable side effect of this therapy is drug-induced liver injury; however, recently, there have been two reports of creatine kinase (CK) elevation in ADPKD patients on tolvaptan treatment. We set out to monitor and determine the actual incidence of CK elevation and evaluate its potential association with other clinical factors.
This is an observational retrospective multicenter study performed in rapidly progressive ADPKD patients on tolvaptan treatment from Barcelona, Spain. Laboratory tests, demographics, treatment dose, and reported symptoms were collected from October 2018 to March 2021.
Ninety-five patients initiated tolvaptan treatment during follow-up. The medication had to be discontinued in 31 (32.6%) patients, primarily due to aquaretic effects (12.6%), elevated liver enzymes (8.4%), and symptomatic or persistently elevated CK levels (3.2%). Moreover, a total of 27 (28.4%) patients had elevated CK levels, with most of them being either transient (12.6%), mild and asymptomatic (4.2%), or resolved after dose reduction (3.2%) or temporary discontinuation (2.1%).
We pre-sent the largest cohort that has monitored CK levels in a real-life setting, finding them elevated in 28.4% of patients. More research and monitoring will help us understand the clinical implications and the pathophysiological mechanism of CK elevation in this population.
常染色体显性多囊肾病（ADPKD）是终末期肾病的最常见遗传原因。目前，托伐普坦是唯一被证明可以延缓疾病进展的治疗方法。这种治疗最显著的副作用是药物引起的肝损伤；然而，最近有两份报告称，托伐普坦治疗的 ADPKD 患者的肌酸激酶（CK）升高。我们着手监测并确定 CK 升高的实际发生率，并评估其与其他临床因素的潜在关联。
这是一项在西班牙巴塞罗那进行的、针对接受托伐普坦治疗的快速进展性 ADPKD 患者的观察性回顾性多中心研究。从 2018 年 10 月至 2021 年 3 月，收集了实验室检查、人口统计学、治疗剂量和报告的症状。
95 名患者在随访期间开始接受托伐普坦治疗。31 名（32.6%）患者因水潴留作用（12.6%）、肝酶升高（8.4%）和症状性或持续性升高的 CK 水平（3.2%）而停用药物。此外，共有 27 名（28.4%）患者的 CK 水平升高，其中大多数患者为一过性升高（12.6%）、轻度无症状（4.2%）或在减少剂量（3.2%）或暂时停药（2.1%）后恢复正常。
我们提出了迄今为止监测 CK 水平的最大队列，发现 28.4%的患者 CK 水平升高。更多的研究和监测将帮助我们了解该人群 CK 升高的临床意义和病理生理机制。
