Therapies for polycystic kidney disease

Curr Opin Pediatr. 2015 Apr;27(2):227-32. doi: 10.1097/MOP.0000000000000202.

Abstract

Purpose of review: All polycystic diseases of the kidney exhibit tubular or saccular cysts. The cysts can either be open to the tubule or isolated sacs that have lost their connections. Polycystic kidney diseases derived from different genetic mutations share basic mechanisms of cytogenesis, formation, and progressive enlargement, involving a cellular organelle called the primary cilium. Given the mechanistic commonalities, this review will focus on the therapeutic approaches currently available or under development that likely apply to all inherited renal cystic diseases.

Recent findings: Recent advances in clinical trials and preclinical experiments have illuminated common signaling pathway involvement.

Summary: Avoidance of nephrotoxic drugs or radio-contrast and maintaining normal BMI are routine preventive measures. Limiting the intake of calories, salt, and protein, together with increased intake of fruits, vegetables, and water are dietary treatments that should be started early in the course of the disease. Potential pharmacological treatments targeting cyst initiation and progression are on the horizon.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Diet, Sodium-Restricted*
  • Directive Counseling
  • Disease Progression
  • Exercise
  • Fruit
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use*
  • Polycystic Kidney Diseases / physiopathology
  • Polycystic Kidney Diseases / therapy*
  • Pravastatin / therapeutic use*
  • Prognosis
  • Renin-Angiotensin System / drug effects*
  • Risk Reduction Behavior
  • Vegetables

Substances

  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Pravastatin