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Biomarkers and surrogate endpoints in kidney disease

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Abstract

Kidney disease and its related comorbidities impose a large public health burden. Despite this, the number of clinical trials in nephrology lags behind many other fields. An important factor contributing to the relatively slow pace of nephrology trials is that existing clinical endpoints have significant limitations. “Hard” endpoints for chronic kidney disease, such as progression to end-stage renal disease, may not be reached for decades. Traditional biomarkers, such as serum creatinine in acute kidney injury, may lack sensitivity and predictive value. Finding new biomarkers to serve as surrogate endpoints is therefore an important priority in kidney disease research and may help to accelerate nephrology clinical trials. In this paper, I first review key concepts related to the selection of clinical trial endpoints and discuss statistical and regulatory considerations related to the evaluation of biomarkers as surrogate endpoints. This is followed by a discussion of the challenges and opportunities in developing novel biomarkers and surrogate endpoints in three major areas of nephrology research: acute kidney injury, chronic kidney disease, and autosomal dominant polycystic kidney disease.

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Acknowledgments

Dr. Hartung is supported by the National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) under Award Number KL2TR000139. The content is solely the responsibility of the author and does not necessarily represent the official view of NCATS or the NIH.

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  1. Division of Nephrology, Children’s Hospital of Philadelphia, 34th and Civic Center Boulevard, Philadelphia, PA, 19104, USA

    Erum A. Hartung

  2. Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, 415 Curie Blvd, Philadelphia, PA, 19104, USA

    Erum A. Hartung

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Hartung, E.A. Biomarkers and surrogate endpoints in kidney disease. Pediatr Nephrol 31, 381–391 (2016). https://doi.org/10.1007/s00467-015-3104-8

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