484 - Refinement Of NS-SIM-PRO And NS-SIM-ObsRO by Cognitive Debriefing of Clinical Outcome Assessment Set (COA) For Nephrotic Syndrome (Prepare-NS) to Measure Edema Impact in Children

Publication Number: 2471.484

Eloise Salmon, University of Michigan Medical School, Ann Arbor, MI, United States; Somayeh Hashemi, University of Michigan Medical School, Ann Arbor, MI, United States; Ashley E. Rahimi, University of Michigan Medical School, Ann Arbor, MI, United States; Rebecca Scherr, University of Michigan Medical School, Ann Arbor, MI, United States; Yan Zhai, University of Michigan Medical School, Ann Arbor, MI, United States; Noelle Carlozzi, University of Michigan Medical School, Ann Arbor, MI, United States; Trivellore Raghunathan, University of Michigan, Whitmore Lake, MI, United States; Courtney Hurt, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Maja Kuharic, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Jin-Shei Lai, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Devin D. Peipert, University of Birmingham, Birmingham, England, United Kingdom

Background: Individuals with nephrotic syndrome (NS) often experience fluid overload (FO), which can negatively affect their health-related quality of life. In collaboration with the Food and Drug Administration (FDA), the initiative "PREPARing a Clinical Outcomes Assessment Set for Nephrotic Syndrome" (Prepare-NS) aims to develop and validate clinical outcome assessments (COAs) for FO in NS, specifically for use in drug development. The COA set will include an observer-reported outcome (ObsRO) for young children and a patient-reported outcome (PRO) for older children and adults. Previous study phases included gap analysis and concept elicitation interviews, which led to the development of draft Swelling Impact Measures (SIMs). This abstract focuses on the cognitive debriefing process for the SIMs involving patients aged 8 to 17 years with NS and parents or caregivers of children aged 2 to 11 years with NS.

Objective: The purpose of the cognitive debriefing interviews was to review and refine the newly drafted Swelling Impact Measures.

Design/Methods: Participants were informed about the study through clinical sites and patient advocacy groups and were recruited at the University of Michigan. Following consent/assent, each cognitive interview lasted approximately 45 minutes. Participants received a survey link through RedCap to complete online. Then, the interviewer reviewed the survey items over Zoom, asking follow-up questions. Items and interview guides were modified iteratively based on the feedback collected. The focus areas included item interpretation, relevance, recall period, comprehensiveness, and meaningful change.

Results: A total of 6 older children and 14 caregivers of young children with NS participated in the interviews. Table 1 provides a summary of their demographic information. The relevance of the assessments was reported as follows: PRO at 76% and ObsRO at 67%. In terms of comprehensibility, the scores were 97% for PRO and 100% for ObsRO. Figure 1 shows key quotations from pediatric participants about use of the word fatigue as well as pain versus discomfort. Figure 2 illustrates the modifications made to the SIMs based on the feedback received from all participants, children and adults.

Conclusion(s): The 21 items selected as candidates for the NS-SIM-PRO and the 19 items chosen for the NS-SIM-ObsRO will proceed to a validation study. During this phase, individuals with NS and current swelling, or their caregivers if they are young children, will complete surveys at enrollment, and again at 3 months and 9 months.

Table 1
Demographic characteristics of pediatric PRO and all ObsRO participants

Figure 1
Quotations from pediatric PRO participants about fatigue and pain vs. discomfort during cognitive debriefing interviews

Figure 2
Modifications made to the SIMs based on feedback from all participants