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

Pankaj Hari¹, Priyanka Khandelwal², Olivia Boyer³, Rajendra Bhimma⁴, Francesco Cano⁵, Martin Christian⁶, Ali Duzova⁷, Kazumoto Iijima⁸, Hee Gyung Kang⁹, Shen Qian¹⁰, Hesham Safouh¹¹, Susan Samuels¹², William E Smoyer¹³, Marina Vivarelli¹⁴, Arvind Bagga², Franz Schaefer¹⁵; IPNA Best Practices and Standards Committee and c4c Pediatric Nephrology Expert Working Group

Affiliations

¹ Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India. pankajhari@hotmail.com.
² Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India.
³ Centre de Référence MARHEA, Institut Imagine, Néphrologie Pédiatrique, Université Paris Cité, Hôpital Necker - Enfants Malades, Paris, France.
⁴ Department of Paediatrics and Child Health, University of KwaZulu-Natal, Durban, South Africa.
⁵ Department of Nephrology, Luis Calvo Mackenna Children's Hospital, University of Chile, Santiago, Chile.
⁶ Nottingham Children's Hospital, Nottingham University Hospitals, Nottingham, UK.
⁷ Division of Pediatric Nephrology, Department of Pediatrics, Faculty of Medicine, Hacettepe University, Ankara, Turkey.
⁸ Department of Pediatrics, Kobe University Graduate School of Medicine, Kobe, Japan.
⁹ Division of Pediatric Nephrology, Department of Pediatrics, Seoul National University Children's Hospital & Seoul National University College of Medicine, Seoul, Korea.
¹⁰ Clinical Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
¹¹ Pediatric Nephrology Unit, Faculty of Medicine, Cairo University, Cairo, Egypt.
¹² Department of Pediatrics, Section of Pediatric Nephrology, Alberta Children's Hospital, University of Calgary, Calgary, Canada.
¹³ The Research Institute at Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
¹⁴ Laboratory of Nephrology and Clinical Trial Center, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
¹⁵ Center for Pediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.

PMID: 39384644
DOI: 10.1007/s00467-024-06543-x

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

Pankaj Hari et al. Pediatr Nephrol. 2025 Mar.

. 2025 Mar;40(3):865-872.

doi: 10.1007/s00467-024-06543-x. Epub 2024 Oct 10.

Authors

Affiliations

¹ Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India. pankajhari@hotmail.com.
² Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India.
³ Centre de Référence MARHEA, Institut Imagine, Néphrologie Pédiatrique, Université Paris Cité, Hôpital Necker - Enfants Malades, Paris, France.
⁴ Department of Paediatrics and Child Health, University of KwaZulu-Natal, Durban, South Africa.
⁵ Department of Nephrology, Luis Calvo Mackenna Children's Hospital, University of Chile, Santiago, Chile.
⁶ Nottingham Children's Hospital, Nottingham University Hospitals, Nottingham, UK.
⁷ Division of Pediatric Nephrology, Department of Pediatrics, Faculty of Medicine, Hacettepe University, Ankara, Turkey.
⁸ Department of Pediatrics, Kobe University Graduate School of Medicine, Kobe, Japan.
⁹ Division of Pediatric Nephrology, Department of Pediatrics, Seoul National University Children's Hospital & Seoul National University College of Medicine, Seoul, Korea.
¹⁰ Clinical Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
¹¹ Pediatric Nephrology Unit, Faculty of Medicine, Cairo University, Cairo, Egypt.
¹² Department of Pediatrics, Section of Pediatric Nephrology, Alberta Children's Hospital, University of Calgary, Calgary, Canada.
¹³ The Research Institute at Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
¹⁴ Laboratory of Nephrology and Clinical Trial Center, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
¹⁵ Center for Pediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.

PMID: 39384644
DOI: 10.1007/s00467-024-06543-x

Abstract

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.

Keywords: Endpoint; Estimated glomerular filtration rate; Kidney failure; Proteinuria; Remission.

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Conflict of interest statement

Declarations. Conflict of interest: OB provides scientific advice or participates in sponsored clinical trials for Alexion, Alnylam, Biocodex, Bristol-Myers Squibb, Glaxo Smith Kline, Purespring, Roche, Sanofi, Takeda, Travere/CSL/Vifor. MV has participated in sponsored clinical trials, provided consulting, participated in advisory boards, or given sponsored lectures for Novartis, Roche, Apellis, Alexion, Vifor, Glaxo, Chinook, Bayer, Biocryst, Travere, Pure Spring. FS has received consulting fees from Roche, Stada, and Bayer and research support from Novartis. The above activities do not influence the content of the current manuscript for all authors.

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

Affiliations

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

Authors

Affiliations

Abstract

Conflict of interest statement

References

Publication types

MeSH terms

Substances