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

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 pa

SUBMITTER: McKay AM 

PROVIDER: S-EPMC9048617 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature