In an article by Roser Torra, MD, PhD, for Medscape, Torra discusses management and treatment options for those who have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD).*

What Is ADPKD?

• ADPKD involves buildup of cysts accompanied by progressive increase in kidney volume.
• The disease is characterized by a fluid collection in or on the kidney called renal cysts.
• In addition to the kidney, the cysts may appear in the liver, pancreas, and other organs.
• ADPKD is the most frequent monogenic human disorder, meaning it can manifest due to a change in a single gene—fibrocystin (PKHD1).
• The disease affects approximately fifteen million people worldwide.
• ADPKD patients have a 50% risk of end-stage kidney disease.
• About half of patients require kidney replacement therapy by age 60.

Treatment

• For a long time, treatment and management strategies for ADPKD were minimal compared to other kidney diseases.
• Recently, clinical trials of the drug Tolvaptan showed a reduced rate of cysts growth and a slower decline in kidney function.  
• The drug affects vasopressin receptor 2 (V2R) in the kidney tubule. Vasopressin is a circulating hormone produced in the hypothalamus of the brain that regulates body fluid concentration by binding to V2R.
• Japan and the European Medical Agency have approved Tolvaptan for the treatment of ADPKD in adults with stage 1 to 3 chronic kidney disease.
• Treatment with Tolvaptan is recommended only in rapidly progressing disease.
• In the USA, the Food and Drug Administration (FDA) has demanded additional results to better evaluate the safety and effectiveness of this medication.

High Blood Pressure (Hypertension)

• A frequent complication of ADPKD is hypertension, which happens in more than half of patients and usually appears before the symptoms of kidney dysfunction.
• Controlling hypertension is the primary and most effective therapy. Effective treatment of hypertension should be started as early as possible to decrease mortality of ADPKD patients.

Liver Cysts

• In adult patients with ADPKD, cysts in the liver may develop later than kidney cysts. About 80% of ADPKD patients 60 years and older have liver cysts.
• Treatment with somatostatin-class drugs has shown preliminary effectiveness.

Lifestyle Modifications

• Patients with ADPKD should avoid violent sports.
• While reducing salt is advised for ADPKD patients with hypertension or kidney failure, no other diet has proven effective.
• Non-hypertensive ADPKD patients with normal kidney function should undergo annual blood and urinary testing and ultrasonography of the kidneys.

Additional information on the treatment and management of ADPKD can be found in Chapter 3 (Treatment and Management of Autosomal Dominant Polycystic Kidney Disease) of the book Polycystic Kidney Disease edited by Li X. 

*Torra, R. (2020, March 24). Polycystic Kidney Disease Treatment & Management. Medscape.