[Autosomal dominant polycystic kidney disease: is the treatment for tomorrow?]

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most frequent Mendelian inherited disorder. It covers 6.1% of incident ESRD patients in France in 2011. Long left untreated, this disease will soon benefit from targeted therapies currently under evaluation. Several molecules have already reached the stage of clinical trials: the evaluation of mTOR inhibitors yielded deceiving results and, more recently, 2 different molecules demonstrated a slight impact on the progression of total kidney volume (TKV): tolvaptan, vasopressin receptor-V2 inhibitor and somatostatin analogues; both of these molecules acting throughout the decrease of intracellular AMPc. The purpose of this review is to briefly describe the signaling pathways involved, then to present both the published and ongoing clinical trials and the promising molecules evaluated in murine models.

Keywords: Analogues de la somatostatine; Autosomal dominant polycystic kidney disease; Chronic kidney disease; Drug therapy; Everolimus; Genetic diseases; Génétique; Inhibiteur de la voie des mTOR; Inhibiteurs des récepteurs V2 de la vasopressine; Insuffisance rénale chronique; Polykystose rénale autosomique dominante; Receptors; Sirolimus; Somatostatin analogs; Thérapeutique; Vasopressin/antagonists and inhibitors.