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Cystogenesis pp 105-121 | Cite as

Clinical Trials and a View Toward the Future of ADPKD

  • Hyunsuk KimEmail author
  • Young-Hwan Hwang
Chapter
  • 889 Downloads
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 933)

Abstract

In light of the advances in the understanding of cystogenesis in clinical syndromes, potential therapeutic targets have been proposed. Among ciliopathies, autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary disease, and is characterized by the progressive enlargement of bilateral renal cysts, resulting in end-stage kidney failure. Progress in genetics and molecular pathobiology has enabled the development of therapeutic agents that can modulate aberrant molecular pathways. Recently, clinical trials using somatostatin analogs and vasopressin receptor antagonists were conducted, and resulted in the approval of tolvaptan in managing kidney disease in some countries. We will summarize the developments of therapeutic agents based on pathogenesis, and discuss recent findings in clinical trials. Moreover, issues such as the timing of the intervention and outcome assessment will be discussed.

Keywords

Polycystic kidney Autosomal dominant Cystogenesis Therapeutics Clinical trials 

Notes

Acknowledgement

The authors would like to express appreciation for the great help of Professors Curie Ahn (Seoul National University Hospital) and Kyubeck Lee (Kangbuk Samsung Hospital), who have devoted themselves to managing patients with ADPKD over the past two decades. We would also like to extend our gratitude to Danang Sarang (www.pkdkorea.co.kr/), a group for patients with ADPKD in Korea, and the PKD Foundation International (http://www.pkdinternational.org/).

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Copyright information

© Springer Science+Business Media Singapore 2016

Authors and Affiliations

  1. 1.Department of Internal MedicineSeoul National University HospitalSeoulSouth Korea
  2. 2.Department of Internal MedicineEulji General HospitalSeoulSouth Korea

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