title: Clinical Trials and a View Toward the Future of ADPKD.
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.
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, 101, Daehak-ro, Jongno-gu, Seoul, 03080, South Korea. keeee@hanmail.net.
authors.forename: Hyunsuk
authors.initials: H
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Eulji General Hospital, 14, Hangeulbiseok-gil, Nowon-gu, Seoul, 01830, South Korea.
authors.forename: Young-Hwan
authors.initials: YH
authors.lastname: Hwang
mesh_terms: D001794:Blood Pressure; D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D058990:Molecular Targeted Therapy; D017063:Outcome Assessment, Health Care; D016891:Polycystic Kidney, Autosomal Dominant / Q000188:drug therapy* / Q000503:physiopathology; D014867:Water / Q000494:pharmacology
chemical_list: D014867:Water
keywords: Autosomal dominant; Clinical trials; Cystogenesis; Polycystic kidney; Therapeutics