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Pediatric Nephrology

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28.04.2022 | Review

Therapeutic trials in difficult to treat steroid sensitive nephrotic syndrome: challenges and future directions

verfasst von: Ashlene M. McKay, Rulan S. Parekh, Damien Noone

Erschienen in: Pediatric Nephrology | Ausgabe 1/2023

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Abstract

Steroid sensitive nephrotic syndrome is a common condition in pediatric nephrology, and most children have excellent outcomes. Yet, 50% of children will require steroid-sparing agents due to frequently relapsing disease and may suffer consequences from steroid dependence or use of steroid-sparing agents. Several steroid-sparing therapeutic agents are available with few high quality randomized controlled trials to compare efficacy leading to reliance on observational data for clinical guidance. Reported trials focus on short-term outcomes such as time to first relapse, relapse rates up to 1–2 years of follow-up, and few have studied long-term remission. Trial designs often do not consider inter-individual variability, and differing response to treatments may occur due to heterogeneity in pathogenic mechanisms, and genetic and environmental influences. Strategies are proposed to improve the quantity and quality of trials in steroid sensitive nephrotic syndrome with integration of biomarkers, novel trial designs, and standardized outcomes, especially for long-term remission. Collaborative efforts among international trial networks will help move us toward a shared goal of finding a cure for children with nephrotic syndrome.

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Titel: Therapeutic trials in difficult to treat steroid sensitive nephrotic syndrome: challenges and future directions
verfasst von: Ashlene M. McKay
Rulan S. Parekh
Damien Noone
Publikationsdatum: 28.04.2022
Verlag: Springer Berlin Heidelberg
Erschienen in: Pediatric Nephrology / Ausgabe 1/2023
Print ISSN: 0931-041X
Elektronische ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-022-05520-6

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Therapeutic trials in difficult to treat steroid sensitive nephrotic syndrome: challenges and future directions

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