Data from animal and human studies suggest that the rate of progression of renal insufficiency
can be retarded with careful control of blood pressure, institution of a low-protein
diet, and the use of lipid-lowering agents. These therapeutic interventions become
important when managing patients with renal insufficiency secondary to autosomal dominant
polycystic kidney disease (PKD) and autosomal recessive polycystic kidney disease,
in which end-stage renal disease is present in nearly 17,000 individuals per year.
Several dietary and pharmacologic intervention strategies including blood pressure
control, dietary modification, and the use of antioxidants as well as lipid-lowering
agents have been studied in humans and animals with PKD in an effort to slow the rate
of renal progression. This article reviews the current understanding of the effectiveness
of these conventional therapies, as well as novel therapies that specifically target
the mediators of cyst formation in PKD using tyrosine kinase inhibitors and gene therapy
in an effort to identify potential strategies for retarding cyst formation and parenchymal
injury in PKD. Current pharmacologic and dietary strategies fail to show any consistent
benefits in preserving renal function and reducing renal injury in human PKD. The
therapeutic potential for exciting new gene therapies and pharmacologic agents designed
to target the pathophysiologic pathways involved in cyst formation are promising.
Randomized, controlled trials in children and adults with early PKD are necessary
to evaluate the effectiveness of these therapeutic interventions.