Introduction
hereditary kidney disorders, ADPKD, impairment of kidney function, Autosomal dominant polycystic kidney disease
ADPKD, KIDNEY FAILURE, DISEASE PROGRESSION, AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common hereditary kidney disorders, characterized by the progressive formation and growth of cysts, leading to impairment of kidney function and eventually kidney failure.Given the phenotypic variability, uncertainty exists regarding the management of children with ADPKD or those at risk because of family history.The management of ADPKD in adults has been changed by the vasopressin V2 receptor antagonist tolvaptan. In the TEMPO 3:4 clinical trial, tolvaptan inhibited the cyst-driven increase in total kidney volume (TKV) and slowed the decline in kidney function in adults at risk of rapid disease progression, with effects on kidney function confirmed in the REPRISE trial.We conducted a phase 3, 1-year, randomized, double-blind, placebo-controlled, multicenter trial in participants with ADPKD aged 4–17 years. The trial objectives were to evaluate the pharmacodynamics, safety, and efficacy of tolvaptan in children and adolescents with ADPKD.