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IPNA consensus definitions for clinical trial outcomes in steroid-resistant nephrotic syndrome

Assessment of the true impact of therapeutic interventions is a challenge in the absence of universal, standardized definitions for clinical trial endpoints in children with kidney diseases. Steroid-resistant nephrotic syndrome (SRNS) is a difficult kidney disease to treat, with unremitting disease...

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Main Authors: Hari, Pankaj (Author) , Khandelwal, Priyanka (Author) , Boyer, Olivia (Author) , Bhimma, Rajendra (Author) , Cano, Francesco (Author) , Christian, Martin (Author) , Düzova, Ali (Author) , Iijima, Kazumoto (Author) , Kang, Hee Gyung (Author) , Qian, Shen (Author) , Safouh, Hesham (Author) , Samuels, Susan (Author) , Smoyer, William E. (Author) , Vivarelli, Marina (Author) , Bagga, Arvind (Author) , Schaefer, Franz (Author)
Format: Article (Journal)
Language:English
Published: March 2025
In: Pediatric nephrology
Year: 2025, Volume: 40, Issue: 3, Pages: 865-872
ISSN:1432-198X
DOI:10.1007/s00467-024-06543-x
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1007/s00467-024-06543-x
Verlag, lizenzpflichtig, Volltext: https://link.springer.com/article/10.1007/s00467-024-06543-x
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Author Notes:Pankaj Hari, Priyanka Khandelwal, Olivia Boyer, Rajendra Bhimma, Francesco Cano, Martin Christian, Ali Duzova, Kazumoto Iijima, Hee Gyung Kang, Shen Qian, Hesham Safouh, Susan Samuels, William E. Smoyer, Marina Vivarelli, Arvind Bagga, Franz Schaefer, on behalf of the IPNA Best Practices and Standards Committee and c4c Pediatric Nephrology Expert Working Group
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Summary:Assessment of the true impact of therapeutic interventions is a challenge in the absence of universal, standardized definitions for clinical trial endpoints in children with kidney diseases. Steroid-resistant nephrotic syndrome (SRNS) is a difficult kidney disease to treat, with unremitting disease progressing to kidney failure. Currently, available therapies result in suboptimal cure rates. Clinical trials with innovative, targeted treatments will likely be conducted for this disease in the foreseeable future. An international consortium of the IPNA Best Practices and Standards Committee and the Pediatric Nephrology Expert Group of the conect4children (c4c) network developed through consensus, standardized, internationally acceptable definitions for trial outcomes for SRNS. The endpoint definitions were formulated for use with urine protein to creatinine ratios and estimated glomerular filtration rates. Definitions of complete remission, partial remission, non-remission of disease, reduction in proteinuria, kidney disease progression, kidney failure, and composite kidney outcome were refined using an iterative process until a consensus was achieved.
Item Description:Online verfügbar: 10.Oktober 2024
Gesehen am 25.04.2025
Physical Description:Online Resource
ISSN:1432-198X
DOI:10.1007/s00467-024-06543-x

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