Estimating risk of rapid disease progression in pediatric patients with autosomal dominant polycystic kidney disease: a randomized trial of tolvaptan.

ABSTRACT:

Background

Tolvaptan preserves kidney function in adults with autosomal dominant polycystic kidney disease (ADPKD) at elevated risk of rapid progression. A trial (NCT02964273) evaluated tolvaptan safety and pharmacodynamics in children (5-17 years). However, progression risk was not part of study eligibility criteria due to lack of validated criteria for risk assessment in children. As risk estimation is important to guide clinical management, baseline characteristics of the study participants were retrospectively evaluated to determine whether risk of rapid disease progression in pediatric ADPKD can be assessed and to identify parameters relevant for risk estimation.

Methods

Four academic pediatric nephrologists reviewed baseline data and rated participant risk from 1 (lowe

SUBMITTER: Mekahli D 

PROVIDER: S-EPMC10942936 | biostudies-literature | 2024 May

REPOSITORIES: biostudies-literature