Federal Grant Award Summary

The Children's Hospital of Philadelphia Research Institute received a $949,759 Project Grant award from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847), effective May 15, 2026, through February 28, 2031. This award funds a real-world comparative effectiveness research study on membranous nephropathy (MN), an autoimmune glomerular disease causing nephrotic syndrome in adults and children. The research applies modern causal inference methods to observational data to evaluate treatment effectiveness, safety outcomes, and the prognostic value of anti-phospholipase A2 receptor antibody (PLA2R-AB) as a biomarker for long-term clinical outcomes.

The project delivers evidence-based treatment guidance by analyzing pooled data from two observational cohort studies (the Nephrotic Syndrome Study Network and CURE Glomerulonephropathy) and electronic health records from two registries (Kidney Research Network and Glomerular Disease Collaborative Network). Deliverables include real-world comparative treatment effectiveness analyses on long-term clinical outcomes, evaluation of PLA2R-AB prognostic value within treatment subgroups, and validation of PLA2R-AB as a surrogate endpoint. Using longitudinal matching, mediation analyses, and survival data analysis methods, this research addresses critical gaps in treatment guidance for membranous nephropathy by providing evidence to inform nephrologist and patient treatment decisions where FDA-approved treatments remain absent and clinical trial evidence is limited.