Treatment of Established ADPKD

Disease-Modifying Pharmacotherapy

The American College of Cardiology recommends tolvaptan as the cornerstone pharmacologic therapy for patients with established ADPKD at risk of rapid progression, combined with blood pressure control targeting systolic <120 mm Hg, increased water intake of 2-3 liters daily, dietary sodium restriction, and preparation for kidney replacement therapy when kidney failure approaches 1, 2
Tolvaptan reduces eGFR decline by 1.3 mL/min/1.73 m² per year and total kidney volume growth by 2.7% 1
Tolvaptan significantly reduces incidence of UTI, kidney stones, hematuria, and kidney pain 1
Patients on tolvaptan require adequate water intake to replace urinary losses for long-term tolerability 2
The Kidney Disease: Improving Global Outcomes (KDIGO) guidelines recommend monitoring liver enzymes closely due to risk of hepatotoxicity 3

The National Kidney Foundation recommends metformin when eGFR ≥30 mL/min/1.73 m² 1, 5
The American Diabetes Association recommends GLP-1 receptor agonist when eGFR <30 mL/min/1.73 m², when metformin not tolerated, or when metformin alone insufficient 1, 5
The Kidney Disease: Improving Global Outcomes (KDIGO) guidelines do not advise SGLT2 inhibitors due to lack of evidence in ADPKD 5, 6

The American Society of Transplantation recommends kidney transplantation, preferably preemptive living-donor transplantation, as the preferred treatment for kidney failure in ADPKD 1, 2, 5
Native nephrectomy should be performed only for specific indications, such as severe symptoms from massively enlarged kidneys, recurrent or severe kidney infection or bleeding, complicated nephrolithiasis, intractable pain, suspicion of renal cell carcinoma, insufficient space for kidney graft, or severe ventral hernia 1, 2, 4, 5, 6
The European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) recommends shared decision-making and hemodialysis prescription same as non-ADPKD patients 1, 2, 5
Peritoneal dialysis is a viable option, but use caution with massive kidney/liver enlargement or abdominal wall hernias 1, 2, 5