What is the management and treatment for polycystic kidney disease (PKD)?

Management and Treatment of Polycystic Kidney Disease (PKD)

The management of polycystic kidney disease should focus on blood pressure control, lifestyle modifications, and targeted pharmacological interventions, with vasopressin antagonists being the primary disease-modifying treatment for patients at risk of rapid progression. 1, 2

Blood Pressure Management

• Standardized blood pressure measurement is recommended for all PKD patients regardless of kidney function, complemented with home or ambulatory monitoring 1
• Renin-angiotensin system inhibitors (ACEIs or ARBs) are first-line treatment for hypertension in PKD 1
• Blood pressure targets:
  • For patients 18-49 years with CKD G1-G2 and BP >130/85 mmHg: target 110/75 mmHg measured by home monitoring 1
  • For patients ≥50 years and/or with CKD G3-G5: systolic blood pressure <120 mmHg measured in office 1
• Avoid any combination of ACEIs, ARBs, and direct renin inhibitors 1
• Resistant hypertension requiring ≥3 medications should be investigated for non-adherence or other causes 1

Lifestyle Modifications

• Promote physical activity of moderate intensity for at least 150 minutes per week and strength training at least twice weekly 3, 1
• Maintain normal weight and healthy diet 3, 1
• Encourage low dietary salt intake to help control blood pressure 3, 1
• Recommend high water intake to avoid dehydration, but evidence for slowing disease progression is limited 3
• Avoid excessive protein intake 3, 4
• Avoid tobacco products, limit alcohol consumption (≤1 drink/day for women, ≤2 drinks/day for men), and avoid excessive caffeine 3, 1

Pharmacological Treatment

Vasopressin Antagonists

• Tolvaptan is the only approved disease-modifying treatment for adults with PKD at risk of rapid progression 1, 2
• Important warnings: can cause serious liver injury requiring regular monitoring, produces copious urination with risk of dehydration, and is contraindicated in patients who cannot sense or respond to thirst 1
• Not routinely recommended for children and young people with ADPKD 3

Other Medications

• Avoid vasopressin analogues (e.g., desmopressin) in PKD patients with enuresis due to potential negative effects on cyst growth 3
• mTOR inhibitors should not be used in PKD due to lack of benefit and important adverse effects 3
• Somatostatin analogues are not recommended due to insufficient evidence 3
• Statins: evidence is mixed with some benefit shown in pediatric studies but not confirmed in adult studies 3

Management of Complications

Pain Management

• Investigate flank, abdominal, or back pain to determine if kidney-related 1
• Treatment sequence:
  1. Non-pharmacological and non-invasive interventions as first option 1
  2. Pharmacological treatment if no relief 1
  3. For pain from dominant cysts: cyst aspiration or sclerotherapy 1
  4. For refractory visceral pain: celiac plexus block or percutaneous renal denervation 1
  5. Nephrectomy reserved for intractable severe pain, typically in advanced kidney disease 1

Kidney Stones and Hematuria

• Medical treatment of kidney stones in PKD should be the same as in the general population 1
• Obstructive kidney stones should be managed in specialized centers 1
• Healthcare professionals should discuss the possibility, causes, and natural history of gross hematuria with patients at diagnosis 1

Urinary Tract Infections

• Do not treat asymptomatic bacteriuria 1
• Use first-line therapy (nitrofurantoin, trimethoprim-sulfamethoxazole, fosfomycin) for uncomplicated symptomatic UTIs 1
• Obtain urine culture before starting antibiotics 1
• Treat acute cystitis episodes with the shortest reasonable duration of antibiotics (generally no more than 7 days) 1
• Investigate recurrent UTIs for underlying predisposition 1

Special Considerations

Pregnancy and Reproductive Issues

• Pregnant women with PKD should be followed by a multidisciplinary team 3, 1
• Blood pressure target during pregnancy: ≤130/85 mmHg 1
• Discontinue ACEIs/ARBs, tolvaptan, and other teratogenic drugs before pregnancy 3, 1
• Low-dose aspirin from weeks 12 to 36 to prevent preeclampsia 1
• Preconception counseling should be offered to both men and women with PKD to discuss options to prevent transmitting PKD to future children 3

Screening and Monitoring

• Total kidney volume is an important marker for disease progression and allows stratification of patients into slow or rapid progression categories 5, 2
• The Mayo Imaging Classification (MIC) stratifies patients according to height-adjusted and age-adjusted kidney volume (classes 1A to 1E) 1
• Regular monitoring of blood pressure, kidney function, and associated complications is essential 1, 2

Psychosocial Support

• Screen for and periodically assess psychosocial issues 3
• Implement education programs to promote self-management 3
• Inform patients about PKD patient organizations and support services 3
• Discuss financial impacts of PKD and help patients avoid unnecessary medical expenses 3

Common Pitfalls and Caveats

• Avoid overreliance on imaging to monitor cyst growth in children, as this has limited impact on management 3
• Be cautious with hormonal contraceptives in women with liver cysts, as estrogen may worsen polycystic liver disease 3, 1
• Consider screening for intracranial aneurysms in patients with family history of aneurysms or subarachnoid hemorrhage 1
• Consider echocardiography in patients with severe or uncontrolled hypertension, heart murmur, or family history of thoracic aortic aneurysm 1