The Treatment of Autosomal Dominant Polycystic Kidney Disease.

BACKGROUND: About one in 2000 persons in Europe suffers from autosomal dominant polycystic kidney disease (ADPKD). The treatment of this disease up to the present has been limited to the management of complications.

METHODS: This review is based on pertinent publications, mostly of the last three years, that were retrieved by a selective search in PubMed.

RESULTS: Kidney volume is probably the most important predictive factor for the loss of renal function. A measurement of kidney size is, therefore, recommended as soon as the diagnosis is made. ADPKD patients under age 30 with a combined kidney volume above 1500 mL and an estimated glomerular filtration rate (eGFR) below 90 mL/min are at high risk of needing kidney-replacement therapy (dialysis or transplantation) within 20 years, even if their renal function is normal. Ultrasonographic follow-up can identify affected persons whose risk for rapid progression is especially high. Currently available evidence reveals that, in patients at risk whose renal function is normal, the maintenance of blood pressure at or below a target value of 110/75 mmHg lessens renal enlargement, albuminuria, and left-ventricular hypertrophy. In another study, the treatment of selected patients with tolvaptan, a vasopressin-2 receptor (V2R) blocker, was found to delay cyst enlargement and the related decline in renal function for three years. It is unclear, however, how long the effect of tolvaptan persists, or whether persons whose renal function is already impaired can benefit from it. The main side effects are marked polyuria and, in rare cases, liver toxicity.

CONCLUSION: In patients with ADPKD, an effort should be made to keep the arterial blood pressure below 120/80 mmHg. In patients at high risk of progression whose renal function is still intact (eGFR > 60 mL/min), strict blood pressure control (< 110/75 mm Hg) is indicated, and possibly V2R blockade with tolvaptan as well. Tolvaptan is an expensive drug, and patients taking it must be carefully monitored for hepatotoxicity.