What are the primary treatment and management recommendations for Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

Management of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

The primary treatment and management of ADPKD should focus on aggressive blood pressure control, lifestyle modifications, and disease-specific therapies to slow progression to kidney failure and manage complications. 1

Blood Pressure Management

Blood pressure control is the cornerstone of ADPKD management:

• Target BP goals:

  • Ages 18-49 with CKD G1-G2 and BP >130/85 mmHg: Target ≤110/75 mmHg (home BP monitoring) 1
  • Ages ≥50 and/or CKD G3-G5: Target systolic <120 mmHg (office measurement) 1

• First-line antihypertensive therapy:

  • Renin-angiotensin system inhibitors (ACEi or ARB) 1
  • Avoid combination of ACEi, ARB, and direct renin inhibitors 1

• Monitoring:

  • Regular standardized office BP measurements
  • Home BP monitoring or ambulatory BP monitoring to complement office readings 1
  • Consider ambulatory BP monitoring for difficult-to-control BP, left ventricular hypertrophy, proteinuria, or declining kidney function with normal office BP 1

• Special considerations:

  • Investigate resistant hypertension (requiring ≥3 drugs) for compliance issues or secondary causes 1
  • Investigate high-grade proteinuria for coexisting kidney disease 1

Pain Management

For kidney pain in ADPKD:

1. Initial approach: Investigate to determine if pain is kidney-related 1
2. Management strategy:
   • Start with nonpharmacologic, noninvasive interventions
   • Progress to pharmacologic treatment if needed
   • For refractory pain, refer to a center of expertise 1
3. Interventional options:
   • Cyst aspiration/sclerotherapy for pain from dominant cysts
   • Celiac plexus block or major splanchnic nerve block
   • Percutaneous renal denervation for refractory chronic visceral pain
   • Spinal cord stimulation for moderate-to-severe refractory pain
   • Nephrectomy only for severe intractable pain with advanced kidney disease 1

Management of Complications

Nephrolithiasis

• Treat as in general population
• Refer obstructing stones to centers of expertise 1

Gout

• No routine treatment for asymptomatic hyperuricemia
• Treat symptomatic gout considering kidney function 1

Hematuria

• Educate patients about possibility, causes, and natural history at diagnosis 1

Urinary Tract Infections

• Do not treat asymptomatic bacteriuria
• Use first-line antibiotics for symptomatic UTIs
• Obtain urine culture before starting antibiotics
• Treat recurrent UTIs with short-duration antibiotics (≤7 days)
• Investigate recurrent UTIs for underlying predisposition
• Obtain blood cultures if upper UTI or cyst infection suspected
• For kidney cyst infection, treat with 4-6 weeks of lipid-soluble antibiotics 1, 2

Lifestyle Modifications

• Sodium intake: Limit to <2000 mg/day 2
• Protein intake: Moderate (0.6-0.8 g/kg/day for CKD stages 3-5) 2
• Weight management: Maintain normal body weight 1, 2
• Hydration: Avoid dehydration; drink to satisfy thirst 1
• Caffeine: Limit to equivalent of four or fewer cups of coffee daily 2
• Alcohol: Limit to ≤1 drink/day for females, ≤2 drinks/day for males 2
• Smoking: Cessation is critical as smoking increases risk of intracranial aneurysm development and rupture 2

Disease-Specific Therapy

Tolvaptan (vasopressin V2 receptor antagonist) is FDA-approved for ADPKD treatment to slow kidney function decline and reduce total kidney volume growth 3. Important considerations:

• Must be stopped prior to pregnancy and not restarted until breastfeeding is completed 1
• Avoid vasopressin analogues for nocturnal enuresis in children with ADPKD 1

Pregnancy and Reproductive Health

• Preconception counseling: Offer to both men and women with ADPKD 1
• Medication management: Discontinue renin-angiotensin system inhibitors, tolvaptan, and other teratogenic drugs before pregnancy 1, 2
• During pregnancy:
  • Follow with multidisciplinary team at expert center
  • Monitor BP, kidney function, and proteinuria
  • Monthly urinalysis; treat positive urine cultures even if asymptomatic
  • BP target ≤130/85 mmHg using pregnancy-safe antihypertensives 1
  • More frequent BP monitoring (preferably weekly home monitoring) for pre-existing or pregnancy-diagnosed hypertension 1

Cardiovascular Management

• Echocardiography for patients with severe/uncontrolled hypertension, heart murmur, symptoms of cardiac dysfunction, or family history of thoracic aortic aneurysm 2
• Screen for intracranial aneurysms in patients with personal history of subarachnoid hemorrhage or positive family history of intracranial aneurysm, subarachnoid hemorrhage, or unexplained sudden death 2

Follow-Up and Monitoring

• Regular BP monitoring
• Periodic assessment of kidney function and total kidney volume
• Individualized intracranial aneurysm rescreening every 5-10 years for high-risk patients with negative initial screening 2

Special Considerations for Children

• Pravastatin may slow increase in height-adjusted total kidney volume in children and young adults with ADPKD 1
• Avoid unnecessary protein restriction in children to prevent malnutrition 1
• Prefer alternatives to vasopressin analogues for nocturnal enuresis 1

Renal Replacement Therapy

When kidney failure occurs:

• Kidney transplantation is preferred, ideally preemptive living-donor transplantation
• Native nephrectomy only considered for specific indications
• Both hemodialysis and peritoneal dialysis are viable options 2