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Establishing core outcomes and minimal clinically important differences for childhood steroid sensitive nephrotic

Cal H Robinson1,2,3, Simon Carter4,5, Nowrin Aman6

  • 1Division of Nephrology, The Hospital for Sick Children, Toronto, ON, Canada. cal.robinson@sickkids.ca.

Pediatric Nephrology (Berlin, Germany)
|October 14, 2025
PubMed

Insights

Researchers established consensus on core outcomes for childhood nephrotic syndrome trials. This includes key disease and patient-reported outcomes, improving future research quality and reporting for steroid-sensitive nephrotic syndrome.

Area of Science:

  • Pediatric Nephrology
  • Clinical Trial Methodology
  • Patient-Reported Outcomes

Background:

  • Limited randomized controlled trials (RCTs) in childhood nephrotic syndrome contribute to global practice variation.
  • Inconsistent outcome reporting and lack of justified minimal clinically important differences (MCIDs) hinder research progress.
  • Need for consensus on core outcomes, assessment timepoints, MCIDs, and desirability of outcome ranking (DOOR) endpoints for childhood steroid-sensitive nephrotic syndrome (SSNS).

Purpose of the Study:

  • Establish consensus on core outcomes for childhood SSNS research.
  • Define assessment timepoints and MCIDs for key endpoints.
  • Identify DOOR endpoints to enhance clinical trial design and interpretation.

Main Methods:

  • Two-stage Delphi consensus survey involving international healthcare providers and Canadian patients/caregivers.
  • Likert scale ratings for potential outcomes and MCID value assignments for relapse and remission.
  • Facilitated workshops and thematic analysis of qualitative data to explore perspectives on trial outcomes and MCIDs.

Main Results:

  • Eighty-one participants (45 providers, 36 patients/caregivers) reached consensus.
  • Three core disease outcomes: relapse rate, relapse-free survival, and relapse occurrence.
  • Median MCIDs: 25% absolute difference in relapse risk (1-year) and 10% absolute difference in remission (2 weeks).
  • Five core patient-reported outcomes (PROs) and DOOR endpoints for relapse and remission achieved consensus.

Conclusions:

  • Consensus achieved on core disease and patient-reported outcomes for childhood nephrotic syndrome research.
  • Findings provide a foundation for improving the quality and consistency of future SSNS trials.
  • Standardized outcomes and MCIDs will enhance interpretability and comparability of research findings.
Abstract

Keywords:
ChildCore outcomesDesirability of outcome rankingMinimal clinically important differenceNephrotic syndrome

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