Treatment of Autosomal Dominant Polycystic Kidney Disease.

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.

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