Abstract: SA-PO705
Preparing a Clinical Outcomes Assessment Set for Nephrotic Syndrome (Prepare-NS): A New Core Set of Clinical Outcome Assessments Focused on Fluid Overload for Nephrotic Syndrome
Session Information
- Glomerular Diseases: Clinical, Outcomes, Trials - III
November 05, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1303 Glomerular Diseases: Clinical‚ Outcomes‚ and Trials
Authors
- Salmon, Eloise, University of Michigan, Ann Arbor, Michigan, United States
- Gipson, Debbie, University of Michigan, Ann Arbor, Michigan, United States
- Carlozzi, Noelle E., University of Michigan, Ann Arbor, Michigan, United States
- Lai, Jin-Shei, Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
- Nachman, Patrick H., University of Minnesota Academic Health Center, Minneapolis, Minnesota, United States
- Selewski, David T., Medical University of South Carolina, Charleston, South Carolina, United States
- Lafayette, Richard A., Stanford University School of Medicine, Stanford, California, United States
- Helm, Kelly, Nephcure Kidney International, King of Prussia, Pennsylvania, United States
- Spino, Cathie, University of Michigan, Ann Arbor, Michigan, United States
- Hurt, Courtney N., Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
- Scherr, Rebecca, University of Michigan, Ann Arbor, Michigan, United States
- Kurtz, Vivian, University of Michigan, Ann Arbor, Michigan, United States
- Wang, Yujie, University of Michigan, Ann Arbor, Michigan, United States
- Shamim, Daniayl, University of Michigan, Ann Arbor, Michigan, United States
- Peipert, John D., Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
Background
Individuals with nephrotic syndrome (NS) experience fluid overload (FO). Partnering with FDA, we are creating and validating Clinical Outcome Assessments (COAs) of Patient Reported Outcomes (PRO) and Observer Reported Outcomes (ObsRO) of FO in NS for use in drug development. The initial project phase established a conceptual model, conducted a gap analysis, and assessed available data.
Methods
Stakeholder groups met, context of use (COU) was defined, and a scoping literature review was conducted. Analysis of existing PROMIS HRQOL data from the NEPTUNE and CureGN cohort studies estimated mean score differences (25,75 CIs) for participants with current vs past edema.
Results
COU includes age > 2 yrs, NS etiology (Figure), persistent or relapsing/remitting NS, and edema, excluding dialysis dependence. 1337 manuscripts were reviewed; 9 met inclusion criteria. Two PROs created for FSGS or FSGS/minimal change disease (MCD) included FO items. No ObsROs identified. Secondary analysis of existing FSGS and MCD qualitative data identified edema location and severity ranges. Pooled cohort analysis showed worse HRQOL with edema in 1,678 participants (1246 adults, 432 children), e.g., adult Physical Function (-5.71 (-8.41, -3.01) and child Mobility (-3.8 (-5.86, -1.73)).
Conclusion
No existing PRO or ObsRO focus on assessment of FO and its impact on patients with NS. Scores on generic HRQOL scales vary with edema but do not gather detailed information on FO, making them insufficient for use in drug development. Protocols for concept elicitation and cognitive debriefing are in progress to fill the identified gaps and inform the creation COAs for FO in NS, inclusive of an ObsRO for young children and PRO for self-report for ages 8+. At project end, the final COA set will be publicly available for use as an endpoint in NS trials.
Funding
- Other U.S. Government Support