Changing the Outcome of a Pediatric Disease: Part II — Current Treatment Options in ADPKD

Purpose of Review: The goal of this article is to review appropriate management of autosomal dominant polycystic kidney disease (ADPKD) with specific focus on interventions in childhood. Recent Findings: Enhanced understanding of the pathophysiologic mechanisms of kidney cystogenesis in ADPKD has led to several recent clinical trials in children and adults. The rationale and efficacy of general clinical measures as well as more focused pharmacologic intervention in ADPKD are considered here, including the potential benefit and risk of specific interventions to affected children. Summary: The complex mechanisms of kidney cystogenesis support a variety of potential therapeutic options to slow growth of total kidney volume in ADPKD, thereby altering the long-term outcome of this disease, including risk of end-stage kidney disease (ESKD). It seems likely that a combination of interventions may ultimately prove to be of most clinical benefit, but we need a means to accurately identify children at highest risk of disease progression to address the risk/benefit ratio. More disease-specific therapies are needed in both children and adults with ADPKD. Ultimately, targeted drug delivery to the kidney epithelial cell may play an important role in best outcomes and drug tolerance. © 2022, The Author(s), under exclusive licence to Springer Nature Switzerland AG.