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

Autor: Hari P; Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India. pankajhari@hotmail.com., Khandelwal P; Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India., Boyer O; Centre de Référence MARHEA, Institut Imagine, Néphrologie Pédiatrique, Université Paris Cité, Hôpital Necker - Enfants Malades, Paris, France., Bhimma R; Department of Paediatrics and Child Health, University of KwaZulu-Natal, Durban, South Africa., Cano F; Department of Nephrology, Luis Calvo Mackenna Children's Hospital, University of Chile, Santiago, Chile., Christian M; Nottingham Children's Hospital, Nottingham University Hospitals, Nottingham, UK., Duzova A; Division of Pediatric Nephrology, Department of Pediatrics, Faculty of Medicine, Hacettepe University, Ankara, Turkey., Iijima K; Department of Pediatrics, Kobe University Graduate School of Medicine, Kobe, Japan., Kang HG; Division of Pediatric Nephrology, Department of Pediatrics, Seoul National University Children's Hospital & Seoul National University College of Medicine, Seoul, Korea., Qian S; Clinical Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China., Safouh H; Pediatric Nephrology Unit, Faculty of Medicine, Cairo University, Cairo, Egypt., Samuels S; Department of Pediatrics, Section of Pediatric Nephrology, Alberta Children's Hospital, University of Calgary, Calgary, Canada., Smoyer WE; The Research Institute at Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA., Vivarelli M; Laboratory of Nephrology and Clinical Trial Center, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Bagga A; Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India., Schaefer F; Center for Pediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
Jazyk: angličtina
Zdroj: Pediatric nephrology (Berlin, Germany) [Pediatr Nephrol] 2024 Oct 10. Date of Electronic Publication: 2024 Oct 10.
DOI: 10.1007/s00467-024-06543-x
Abstrakt: 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.
(© 2024. The Author(s), under exclusive licence to International Pediatric Nephrology Association.)
Databáze: MEDLINE
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