Kidney cysts in ADPKD 

ADPKD causes fluid-filled sacs called cysts to form in the kidneys. These cysts damage and enlarge the kidneys.

ADPKD is a progressive disease, meaning it gets worse over the years. By their late 50s, about half of all people with ADPKD develop kidney failure and need dialysis long term or a kidney transplant.

The only medication available to slow down the progression of ADPKD is tolvaptan.

Tolvaptan (brand name Jinarc®) can slow the progression of ADPKD in adults. It works by blocking the receptor for a hormone called vasopressin in your body. This slows the speed at which kidney cysts grow and helps to keep your kidneys functioning for longer.

Tolvaptan is thought to work best when the kidneys are enlarging quickly yet still filtering blood efficiently (meaning they can do at least 30% of the work that a healthy kidney can). Tolvaptan is taken as a tablet twice a day.

Your kidney specialist (nephrologist) can explain whether tolvaptan is suitable for you and talk you through the benefits and risks. In the UK, tolvaptan is not recommended for everyone with ADPKD.

To be eligible, you need to:

• be over 18 years old
• have ADPKD that’s progressing rapidly now or is predicted to progress rapidly in the future
• have stage 2 or 3 kidney disease (or stage 1, 2 or 3 in Scotland) at the time you start treatment

The stage of your kidney disease is estimated by blood tests. Your kidney specialist can explain what stage of disease you have and how quickly your ADPKD might progress. You might need to have further tests over the next 6 or 12 months before your doctor can estimate the speed at which your ADPKD is progressing.

If your kidney function is good and your disease is progressing very slowly, your doctor might say that you don’t need tolvaptan yet. If your kidneys are already failing you may be offered a different treatment (for example, dialysis or a kidney transplant).

3 years of treatment with tolvaptan was tested in a clinical trial of people with ADPKD who had mild kidney disease (stage 1 or 2). Its effects were compared with those of a placebo (a pill without any medicine in it). On average, the people taking tolvaptan had:

• less rapid growth in kidney size
• a slower drop in kidney function

A second trial found that tolvaptan also works in people with ADPKD who have mild-to-moderate kidney disease (stage 3).

The benefits of tolvaptan seem to last for at least 10 years in people who continue to take it.

The most common side effects of tolvaptan include:

• being thirsty and having to drink more water
• needing to pee more often in the day and night
• headaches and dizziness
• diarrhoea
• a dry mouth
• feeling tired

In a clinical trial, around 2 in every 10 patients (20%) stopped taking tolvaptan because of the side effects. The side effect that bothered people most often was needing to pee (and drink) a lot.

Most people who are able to keep taking tolvaptan find it doesn’t affect their quality of life very much.

Tolvaptan can affect the liver but this is not common and reverses on stopping the drug. People taking tolvaptan have regular monthly blood tests to check their liver function.

For a full list of possible side effects, please ask your doctor or check the patient information leaflet that comes with tolvaptan.

If your ADPKD is causing symptoms your kidney specialist or other doctor may recommend medicines to reduce these. Medicines can also help to reduce your chance of future health problems, such as cardiovascular disease.

Medicines for ADPKD symptoms include:

• blood pressure tablets to lower your blood pressure if it is high
• antibiotics to treat an infection in the bladder or kidneys (urinary tract) or liver cysts
• pain killers to treat pain caused by enlarged kidneys

If you have a high risk of cardiovascular disease (which might be partly due to ADPKD), your doctor might recommend additional medicines. These include lipid-lowering drugs (statins) to reduce your cholesterol, and antiplatelet drugs (such as aspirin or clopidogrel) to reduce your risk of a stroke or heart attack.

Whether you need any of these medicines will depend on the symptoms you have. Your doctor or specialist will explain which treatments are best for you and why.

If you’re nearing kidney failure, your kidney specialist will discuss the options of dialysis and transplant with you. For more information, see our factsheet on Progression.

If you have ADPKD and reduced kidney function, some drugs might not be suitable for you. You might also need to take some medicines at a lower dose than standard. Your doctor can advise you on this.

Before taking any medicine (including ones you can buy without prescription) check with your kidney specialist, GP or pharmacist that the medicine is safe for you.

If you have kidney disease, you should only use these medicines if your doctor advises you to:

• non-steroidal anti-inflammatory pain killers such as ibuprofen (Nurofen® Advil® , Brufen® and other brands) and diclofenac
• treatments for indigestion that contain potassium, aluminium, sodium or magnesium
• a heartburn medication called cimetidine
• decongestants such as pseudoephedrine (Brand name Sudafed®).

For more information on medicines to avoid if you have kidney disease, see Over-the-Counter Medicines by Kidney Care UK.

Some medicines may not be safe because:

• Your kidneys won’t clear the medicine out of your body quickly enough, which could lead to side effects.
• The medicine might damage your kidneys.
• The medicine might not work properly.
• Some medicines cannot be taken at the same time as others.

Although there are certain medicines you should avoid, this doesn’t mean you have to cope with untreated symptoms. Ask your doctor which medicines you can use instead.