Autosomal dominant polycystic kidney disease (ADPKD) is a chronic systemic disease that affects all races and ethnicities. It is the fourth leading cause of end-stage kidney disease, and it has a heterogenous phenotype ranging from mild to severe disease. Identifying patients with ADPKD who are at risk of rapid progression can guide therapeutic decisions. Several tools to predict disease severity are available, based on features such as total kidney volume from magnetic resonance imaging, PKD genotype, eGFR trajectory, and the occurrence of hypertension and urologic complications early in life. Over the past decade, more evidence has emerged regarding optimal ADPKD management. The HALT PKD trial supported intensive blood pressure control in patients younger than 50 years of age with preserved kidney function. A healthy lifestyle, including maintaining a healthy weight, salt restriction and smoking cessation, is likely to be beneficial. Tolvaptan, the only disease-modifying agent for ADPKD patients that are at risk of rapid progression, is gaining wider use, but is still limited by its side-effects. This is an exciting time for the ADPKD community as multiple promising interventions are in the pipeline and being investigated.

中文翻译：

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常染色体显性遗传性多囊肾病的治疗。


常染色体显性遗传性多囊肾病 （ADPKD） 是一种影响所有种族和民族的慢性全身性疾病。它是终末期肾病的第四大原因，具有从轻度到重度疾病的异质性表型。识别有快速进展风险的 ADPKD 患者可以指导治疗决策。根据磁共振成像的肾脏总体积、PKD 基因型、eGFR 轨迹以及生命早期高血压和泌尿系统并发症的发生等特征，有几种工具可用于预测疾病严重程度。在过去十年中，出现了更多关于最佳 ADPKD 管理的证据。HALT PKD 试验支持对 50 岁以下肾功能保留的患者进行强化血压控制。健康的生活方式，包括保持健康的体重、限制盐分和戒烟，可能是有益的。托伐普坦是 ADPKD 患者唯一有快速进展风险的疾病调节剂，正在获得更广泛的使用，但仍受到其副作用的限制。对于 ADPKD 社区来说，这是一个激动人心的时刻，因为多种有前途的干预措施正在酝酿中并正在研究中。