Recent Advances in the Management of Autosomal Dominant Polycystic Kidney Disease

Clin J Am Soc Nephrol. 2018 Nov 7;13(11):1765-1776. doi: 10.2215/CJN.03960318. Epub 2018 Jul 26.


Autosomal dominant polycystic kidney disease (ADPKD), the most common monogenic cause of ESKD, is characterized by relentless development of kidney cysts, hypertension, and destruction of the kidney parenchyma. Over the past few years, major advancements in diagnosing, prognosticating, and understanding the pathogenesis and natural course of the disease have been made. Currently, no kidney disease is more suitable for nephron-protective strategies. Early nephrology referral and implementation of these strategies may have a substantial effect. Total kidney volume is a good prognostication marker and allows stratification of patients into slow or rapid progressing disease, with implications for their management. Measurement of total kidney volume, disease stratification, and prognostication are possible using readily available tools. Although some patients require only monitoring and basic optimized kidney protective measures, such as rigorous BP control and various lifestyle and dietary changes, others will benefit from disease-modifying treatments. Vasopressin V2 receptor antagonists, a likely disease-modifying treatment, has been approved in several countries and recently by the US Food and Drug Administration; other therapies, such as somatostatin analogs and other novel agents, are currently in clinical trials. The purpose of this article is to present our views on the optimal management to delay kidney disease progression in ADPKD.

Keywords: ADPKD; Antidiuretic Hormone Receptor Antagonists; Cysts; Dietary Sodium; Disease Progression; Kidney Failure, Chronic; Nephrons; Polycystic Kidney, Autosomal Dominant; Receptors, Vasopressin; Referral and Consultation; Somatostatin; United States Food and Drug Administration; blood pressure; hypertension; vasopressin.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antidiuretic Hormone Receptor Antagonists / therapeutic use*
  • Diet*
  • Disease Progression
  • Humans
  • Hypertension / drug therapy*
  • Kidney / pathology*
  • Organ Size
  • Organism Hydration Status
  • Polycystic Kidney, Autosomal Dominant / drug therapy
  • Polycystic Kidney, Autosomal Dominant / pathology*
  • Polycystic Kidney, Autosomal Dominant / therapy*
  • Prognosis
  • Somatostatin / analogs & derivatives
  • Somatostatin / therapeutic use


  • Antidiuretic Hormone Receptor Antagonists
  • Somatostatin