Evolving Insights Into Treatment of Autosomal Dominant Polycystic Kidney Disease

Arlene B. Chapman, MD


April 08, 2021

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney condition. It is caused by mutations in either the PKD1 or PKD2 gene. Although patients with PKD2 mutations experience milder disease than their counterparts, both groups are burdened with progressive illness. ADPKD results in end-stage renal disease by age 60 in approximately half of patients.

In this ReCAP, Dr Arlene Chapman, chief of nephrology at the University of Chicago, explains how clinicians use renal imaging to stratify risk for progression to renal failure. Depending on a patient's risk level, nephrologists prescribe therapeutic interventions and lifestyle changes to slow disease progression and avoid dialysis or transplant.

New and emerging therapies for ADPKD are beginning to arrive. Dr Chapman reports that high-risk patients will benefit from the vasopressin V2 receptor antagonist tolvaptan, which is proven to both reduce the rate of cyst growth by about 40% and reduce the rate of loss of kidney function over 3 years. More options are currently under investigation.


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