Treatment

• Lisa began therapy with tolvaptan in 2020; she continues monitoring of her hepatic transaminases every 3 months and has had no abnormalities.

• Although Lisa is relatively young, proactive treatment of early ADPKD was shown to be beneficial in an outcome prediction model, potentially delaying the mean age of ESKD by 5 years (5).

 

Management of Lisa’s ADPKD 

• Lisa should be monitored for renal and nonrenal manifestations such as hematuria, infection, nephrolithiasis, pain, and vascular conditions.

• Lisa had been maintaining her blood pressure goal, hydration, managing her hypercholesterolemia, and moderating her sodium and protein intake (4).

• Lisa has reported moderate symptoms of anxiety. Follow-up should include repeat screening with the GAD-7. Depression and anxiety are common in patients with ADPKD and are associated with a diminished quality of life (6).  

• The ADPKD Disease Impact Scale (ADPKD-IS) can be used to evaluate the impact of ADPKD on patients’ quality of life (7).

• While most patients with hepatic cystic disease remain asymptomatic, she should be monitored for potential mechanical complications (8).

• Despite cyst growth, liver parenchyma usually remains functional, and most complications are from enlargement of the liver, such as pain, abdominal distension, skin stretching, back pain, dyspepsia, etc. (8).

• Tolvaptan does not treat liver cysts, but it has been shown to be beneficial in some patients, potentially because the reduction of cAMP inhibits autophagy and proliferation of cholangiocytes (9,10). 

 

Ongoing Care

• Imaging for monitoring of TKV is usually performed every 3-5 years (11). While risk class assignment is often stable, some patients who receive disease modifying treatment may drop to a lower class (12).

• Estimates from clinical trials have shown that treatment can potentially delay ESKD by 1 year for every 4 years of treatment (5).