Combination drug versus monotherapy for the treatment of autosomal dominant polycystic kidney disease

Expert Opin Pharmacother. 2016 Oct;17(15):2049-56. doi: 10.1080/14656566.2016.1232394. Epub 2016 Sep 21.

Abstract

Introduction: Despite progress in the understanding of pathogenetic mechanisms of organ cyst formation in autosomal dominant polycystic kidney disease, current treatment methods are insufficient. Experimental studies and clinical trials target at inhibition of cysts development and to slowing CKD progression.

Areas covered: The purpose of this analysis is to overview available literature regarding treatment of ADPKD. The most important recent events concerning ADPKD treatment are: the results of TEMPO 3/4 study and the registration of tolvaptan in the treatment of patients with CKD stage I-III and rapidly progressive ADPKD by EMA. ERA-EDTA recommendations for use of tolvaptan in ADPKD of 2016 will be useful for the identification of patients with rapid progression of disease who will benefit most from treatment. Clinical trials concerning inhibitors of mTOR and SSAs have not delivered convincing evidence of their effectiveness. Usefulness of statins in ADPKD require confirmation in adults. The HALT-PKD study confirmed that inhibition of RAA system slows progression of ADPKD.

Expert opinion: Current treatment of ADPKD involves: the optimization of life style and combined pharmacological treatment with ACE inhibitors or angiotensin receptor blockers, statins (patients with lipid disorders and cardiovascular disease) and tolvaptan (patients with stage I-III CKD and rapidly progressive ADPKD).

Keywords: ACEi; ADPKD; ARB; cyst formation; somatostatin analogues; statins; tolvaptan.

Publication types

  • Comparative Study
  • Review

MeSH terms

  • Adult
  • Angiotensin Receptor Antagonists / therapeutic use
  • Angiotensin-Converting Enzyme Inhibitors / therapeutic use
  • Benzazepines / administration & dosage
  • Benzazepines / therapeutic use*
  • Disease Progression
  • Drug Therapy, Combination
  • Humans
  • Polycystic Kidney, Autosomal Dominant / drug therapy*
  • Renal Insufficiency, Chronic / drug therapy
  • TOR Serine-Threonine Kinases / antagonists & inhibitors
  • Tolvaptan

Substances

  • Angiotensin Receptor Antagonists
  • Angiotensin-Converting Enzyme Inhibitors
  • Benzazepines
  • Tolvaptan
  • MTOR protein, human
  • TOR Serine-Threonine Kinases