Treatment algorithm

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups: see disclaimer

ACUTE

confirmed autosomal-dominant polycystic disease

1st line – renoprotective lifestyle measures

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
1st line – 

renoprotective lifestyle measures

Renoprotective strategies for all patients are healthy lifestyle choices such as maintenance of optimal weight, regular cardiovascular exercise, and avoidance of smoking.[1][4]

Plus – tolvaptan

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with risk of rapidly progressing disease
Plus – 

tolvaptan

Treatment recommended for ALL patients in selected patient group

Tolvaptan is recommended to slow kidney function decline in adults at risk of rapidly progressing autosomal-dominant PKD (ADPKD).[70]

There is international variance in guidance for the assessment of ADPKD progression.[70][71] The Mayo Clinic imaging classification is a commonly used prognostic tool.[44] In patients with ADPKD with disease in Mayo Clinic imaging classes 1C to 1E, rapid disease progression is likely and treatment with tolvaptan is indicated.[35][71]

Primary options

tolvaptan: 45 mg orally once daily in the morning and 15 mg 8 hours later initially, increase to 60 mg once daily in the morning and 30 mg 8 hours later, and then 90 mg once daily in the morning and 30 mg 8 hours later at weekly intervals according to response

Plus – antihypertensive therapy

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with hypertension
Plus – 

antihypertensive therapy

Treatment recommended for ALL patients in selected patient group

Early detection and rigorous treatment of hypertension is renoprotective.[1][4]

Target blood pressure (BP) should be guided by the HALT polycystic kidney disease trials, which examined BP control in early and late autosomal-dominant PKD (ADPKD) in 2 separate randomized controlled trials.[76][77]

One of the studies compared rigorous BP control (i.e., 95/60 mmHg to 110/75 mmHg) with standard BP control (i.e., 120/70 mmHg to 130/80 mmHg) using an ACE inhibitor and/or angiotensin-II receptor antagonist in patients with ADPKD ages 15 to 49 years who were at risk of progressing to end-stage renal disease. The study found that the annual percentage increase in total kidney volume was lower in the rigorous BP control group (i.e., 95/60 mmHg to 110/75 mmHg). The rate of change in estimated glomerular filtration rate (GFR) was similar in both groups. Left-ventricular-mass index and urinary albumin excretion was reduced in the rigorous BP control group.[76] In a study of patients with more advanced chronic kidney disease (i.e., GFR 25-60 mL/minute), monotherapy with an ACE inhibitor is associated with good BP control in most patients.[77] Evidence does not support the use of dual angiotensin blockade in patients with ADPKD.[76][77]

First-line drug therapy should be with either an ACE inhibitor or an angiotensin-II receptor antagonist.[1] Use of these drug classes increases renal plasma flow in these patients, and offers cardioprotective and renoprotective effects in early ADPKD.[78][79]

Calcium-channel blockers are not considered antihypertensives of choice.

Choice of drug therapy should also be tailored to any specific comorbidity.[31] Noncardioselective beta-blockers with selective alpha-blocking properties (e.g., labetalol or carvedilol) are usually effective. Beta-blockers may be the first choice in a patient with aortic aneurysms, coronary heart disease, or cardiac arrhythmias.

In most cases, diuretics are not needed to control hypertension in ADPKD; however, diuretics may be needed in patients with congestive heart failure and volume overload or resistant hypertension.

Treatment is lifelong.

Dose should be started low and increased gradually according to response.

Primary options

captopril: 12.5 to 150 mg orally three times daily

OR

enalapril: 10-40 mg orally once daily

OR

perindopril erbumine: 4-16 mg orally once daily

OR

ramipril: 2.5 to 20 mg orally once daily

OR

benazepril: 5-80 mg orally once daily

OR

losartan: 25-100 mg orally once daily

OR

candesartan cilexetil: 8-32 mg orally once daily

OR

valsartan: 40-320 mg orally once daily

OR

irbesartan: 150-300 mg orally once daily

OR

telmisartan: 20-80 mg orally once daily

OR

olmesartan medoxomil: 20-40 mg orally once daily

Secondary options

labetalol: 100-400 mg orally twice daily

OR

carvedilol: 6.25 to 25 mg twice daily

Tertiary options

metoprolol tartrate: 100-450 mg/day orally (immediate-release) given in 2-3 divided doses

OR

nebivolol: 5-40 mg orally once daily

OR

hydrochlorothiazide: 12.5 to 25 mg orally once daily

OR

furosemide: 20-40 mg orally once daily

Plus – antibiotic therapy

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with urinary tract infection
Plus – 

antibiotic therapy

Treatment recommended for ALL patients in selected patient group

Urinary tract infections have increased morbidity in patients with autosomal-dominant PKD. They should be treated promptly according to cultures and current treatment guidelines. See our topics Urinary tract infection in women, Urinary tract infection in men, and Urinary tract infection in children.

If the infection relapses after completing antibiotics, complications such as obstruction, cyst infection, or infected stones need to be excluded.

When indicated, urinary tract instrumentation should be done under prophylactic antibiotic coverage before, and for 24 hours after, the procedure.

Plus – antibiotic therapy

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with infected renal cyst
Plus – 

antibiotic therapy

Treatment recommended for ALL patients in selected patient group

Infected renal cysts should be treated with antibiotics first-line.[80]

Fluoroquinolones accumulate in cysts and are considered the antibiotics of choice.[31][80] Trimethoprim/sulfamethoxazole also has good cyst penetration and may be a second-line option.[31]

Fluoroquinolones have been associated with serious, disabling, and potentially irreversible adverse effects, including tendonitis, tendon rupture, arthralgia, neuropathies, and other musculoskeletal or nervous system effects.[81] Warnings have also been issued about the increased risk of aortic dissection, significant hypoglycemia, and mental health adverse effects in patients taking fluoroquinolones.[82][83]

Treatment course is usually 4 to 6 weeks.

Primary options

ciprofloxacin: 250-500 mg orally twice daily; 200-400 mg intravenously twice daily

OR

levofloxacin: 500-750 mg orally/intravenously once daily

Secondary options

sulfamethoxazole/trimethoprim: 160 mg orally twice daily

More

Consider – cyst drainage

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with infected renal cyst
Consider – 

cyst drainage

Treatment recommended for SOME patients in selected patient group

Percutaneous or surgical cyst drainage should be considered if there is no prompt response to treatment with antibiotics. Drainage of large infected cysts should also be considered if there is fever (>100.4°F [>38°C] for >3 days), abdominal pain (particularly a palpable area of renal pain), increased C-reactive protein (>50 mg/L), and the absence of any significant recent intracystic bleeding (based on the results of a computed tomography scan) or other causes of fever.[84]

Consider – nephrectomy

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with infected renal cyst
Consider – 

nephrectomy

Treatment recommended for SOME patients in selected patient group

Severe infections that are resistant to drainage and/or antibiotic therapy may require nephrectomy.[1]

Plus – treatment of underlying cause and supportive care

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with renal pain
Plus – 

treatment of underlying cause and supportive care

Treatment recommended for ALL patients in selected patient group

Cyst hemorrhage, renal infection, stones, or tumors cause renal pain and should be investigated and treated. A stepwise approach to pain management is recommended in patients where no reversible cause can be found.

Bed rest is often helpful. Patients with hematuria should be advised to drink large volumes of fluid and avoid physical activity.

Analgesic therapy includes acetaminophen or opioid analgesics for acute or severe pain. Nonsteroidal anti-inflammatory drugs should generally be avoided but may be used for short periods of time to treat acute pain in patients with good renal function.

Acetaminophen may be used in combination with other analgesics. Tramadol is useful for moderate pain. Oxycodone should not be used for prolonged daily use, but reserved for rescue treatment.

Adjuvant therapies including tricyclic antidepressants, gabapentin, or pregabalin may also be tried, but only under specialist guidance.

Primary options

acetaminophen: 325-1000 mg orally every 4-6 hours when required, maximum 4000 mg/day

Secondary options

ibuprofen: 200-400 mg every 4-6 hours when required, maximum 2400 mg/day

OR

naproxen: 250-500 mg every 12 hours when required, maximum 1250 mg/day

OR

tramadol: 50-100 mg orally every 4-6 hours when required, maximum 400 mg/day

Tertiary options

oxycodone: 2.5 to 5 mg orally (immediate-release) every 6 hours when required

Consider – surgical intervention

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with renal pain
Consider – 

surgical intervention

Treatment recommended for SOME patients in selected patient group

Considered for the management of cyst complications when conservative measures fail.[87]

Cysts are aspirated under computed tomography guidance, with sclerosing drugs used in some patients to prevent fluid reaccumulation. If there are multiple cysts, laparoscopic or surgical cyst fenestration or decortication may be used.[85] Despite a potential role in blood pressure management, cyst decortication has not been definitively shown to alleviate hypertension in patients with autosomal-dominant PKD. Renal function also does not appear to improve following surgery. Patients with compromised baseline renal function appear to be at an increased risk of further deterioration in renal function after cyst decortication. Improvement in pain symptoms appears to be transient, lasting only weeks to months. Therefore, repeat procedures or alternative approaches may be necessary.[86]

Laparoscopic or thoracoscopic renal denervation is considered in some situations.[87]

Patients with cyst hemorrhage and a decrease in hematocrit may require transfusion and, if bleeding persists, angiography with embolization.

In very rare situations, laparoscopic or retroperitoneoscopic unilateral or bilateral nephrectomy is a last-resort option reserved for patients with renal pain who have end-stage renal disease, or in preparation for patients who meet criteria for renal transplant.

Plus – urinary alkalinization + analgesia

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with nephrolithiasis
Plus – 

urinary alkalinization + analgesia

Treatment recommended for ALL patients in selected patient group

Stone type influences management, but potassium citrate is indicated for 3 types of stones seen in autosomal-dominant PKD: uric acid stones, hypocitraturic calcium oxalate nephrolithiasis, and distal acidification defects.[31][88]

Appropriate analgesia should be given. Hospitalization and administration of intravenous fluids may be appropriate.

Urology evaluation may be necessary for symptomatic stones.

See our topic Nephrolithiasis for further details.

Consider – surgical intervention

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with nephrolithiasis
Consider – 

surgical intervention

Treatment recommended for SOME patients in selected patient group

Surgical intervention is indicated in obstructing ureteric calculus, severe pain, and patient inability to maintain hydration.

The choice of intervention depends on several factors, including location of the renal stone within the urinary tract, the clinical presentation (and its urgency), stone characteristics, and other complicating coexisting or intercurrent factors.[89][90]

Extracorporeal shock wave lithotripsy and percutaneous nephrolithotomy can often be performed without a greatly increased risk of complications.[1]

Flexible ureteroscopy with laser fragmentation may also be considered.[1]

Consider – surgical intervention ± antibiotics

Back
ACUTE
|
confirmed autosomal-dominant polycystic disease
|
with symptomatic polycystic liver disease
Consider – 

surgical intervention ± antibiotics

Treatment recommended for SOME patients in selected patient group

Most patients with autosomal-dominant PKD will have liver cysts, but only a minority will be symptomatic.[37]

Symptomatic patients may need interventions to reduce cyst volume and liver size (e.g., cyst drainage, liver resection with cyst fenestration).[37] Choice of procedure is dictated by anatomy and cyst distribution.

Antibiotics are required in patients with infected cysts (diagnosis of hepatic cyst infection may be aided by positron emission tomography scan).[1][58]

Patients with severe disease should be referred to a specialty center for liver resection or transplant.

ONGOING

end-stage renal disease

1st line – renal transplant

Back
ONGOING
|
end-stage renal disease
1st line – 

renal transplant

Renal transplantation is the treatment of choice for end-stage renal disease in autosomal-dominant PKD (ADPKD).[31] Living donor transplant is the preferred option. Cadaveric donor transplant is a second-line option. In the US, current United Network for Organ Sharing rules permit listing on deceased donor renal transplant lists for medically fit individuals with glomerular filtration rate <20 mL/minute.

Sirolimus immunosuppression has been shown to decrease native kidney and liver volumes in a small study of ADPKD patients who had undergone kidney transplant.[98] These observations are too preliminary for recommendation of sirolimus immunosuppression in ADPKD patients.

2nd line – dialysis

Back
ONGOING
|
end-stage renal disease
2nd line – 

dialysis

Dialysis is a second-line option in patients with autosomal-dominant PKD (ADPKD) and end-stage renal disease (ESRD). Patients should have a functioning permanent access at the time of dialysis therapy initiation, and vascular mapping should be completed in all patients before placement of vascular access.

Hemodialysis is preferred over peritoneal dialysis in this patient group, as large kidney size will not permit adequate volumes of dialysis fluid instills, and risk of peritonitis and inguinal or umbilical hernias is increased. However, cyst infections can occur in hemodialysis patients as a result of hematogenous seeding.

The outcome of maintenance hemodialysis in patients with ADPKD is similar to other patient groups, although prevalence of renal pain, hematuria, and renal infection is higher in ADPKD patients. These patients tend to do better on dialysis than patients with ESRD due to other causes.[97]

back arrow

Choose a patient group to see our recommendations

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups. See disclaimer

Use of this content is subject to our disclaimer