TOP 44 - Clinical and Demographic Predictors of Treatment Outcomes and Disease Progression in Pediatric Steroid Resistant Nephrotic Syndrome

Publication Number: 1803.TOP 44

Priyanka Chati, Lurie Children's, Chicago, IL, United States; Grace Y. Lee, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Margaret Fain, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Joseph Fishbein, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States; Vidhi Dalal, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States

Background: Nephrotic syndrome (NS) is one of the most common glomerular disorders in children and one of the leading causes of pediatric chronic kidney disease (CKD). The majority of pediatric cases are classified as idiopathic nephrotic syndrome (INS), for which glucocorticoids remain the first-line therapy. While most children respond favorably and achieve complete remission, approximately 10–15% exhibit steroid resistance, which is associated with a higher risk of multi-drug resistance and progression to end stage kidney disease (ESKD). Currently, no validated biomarkers or clinical tools exist to support early risk stratification of INS. Although prior studies have investigated factors associated with steroid responsiveness, data remain limited regarding children with steroid-resistant nephrotic syndrome (SRNS).

Objective: This study aims to identify clinical and demographic patient factors present at diagnosis that are associated with response to non-steroidal immunosuppressive medications and long-term outcomes in patients with SRNS. Future studies will validate these results in larger cohorts through the use of publicly available datasets to establish baseline patient characteristics that can facilitate risk stratification, personalize therapeutic approaches, and improve prognostication in SRNS.

Design/Methods: This is a single-center retrospective study of SRNS patients aged 1-18 years evaluated at Ann & Robert H. Lurie Children’s Hospital of Chicago (LCH) from 2010 to 2025. The study was reviewed by the LCH Institutional Review Board (IRB) and deemed exempt from ongoing review. Clinical and demographic data, laboratory trends, treatment responses, and outcomes were extracted from the electronic medical records of 373 patients with NS. We are currently analyzing the data to identify SRNS patients from this initial cohort. We will then determine how baseline characteristics for these SRNS patients associate with responsiveness to non-steroidal immunosuppressive medications, relapse patterns, and progression to CKD. Baseline variables will be summarized descriptively. Factors associated with treatment response will be analyzed using logistic regression to estimate odds ratios (ORs) and 95% confidence intervals (CIs). Multivariable models will adjust for confounders including age and sex. Categorical predictors will be analyzed in comparison to a reference group, while continuous variables will be assessed per unit increase. A p-value of < 0.05 will be considered statistically significant.