67 - Development of an observer reported outcome for children with nephrotic syndrome as part of "PREPARing a clinical outcomes assessment set for Nephrotic Syndrome" (PREPARE-NS)

Sunday, April 30, 2023

3:30 PM – 6:00 PM ET

Poster Number: 67
Publication Number: 67.351

Eloise Salmon, University of Michigan Medical School, Ann Arbor, MI, United States; Noelle E. Carlozzi, University of Michigan Medical School, Ann Arbor, MI, United States; Kayla Sifre, University of Michigan Medical School, Ann Arbor, MI, United States; Shawn P. Sullivan, University of Michigan, Laurel, MD, United States; Jin Shei Lai, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Cathie Spino, University of Michigan, Ann Arbor, MI, United States; Yujie Wang, University of Michigan Medical School, Ann Arbor, MI, United States; Tina Creguer, University of Michigan Medical School, Ann Arbor, MI, United States; Courtney N. Hurt, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States; Rebecca Scherr, University of Michigan Medical School, Ann Arbor, MI, United States; Kelly Helm, NephCure, Colorado Springs, CO, United States; Richard Lafayette, Stanford University School of Medicine, Stanford, CA, United States; Patrick H.. Nachman, University of Minnesota, Minneapolis, MN, United States; David Selewski, Medical University of South Carolina, Mount Pleasant, SC, United States; Debbie S. Gipson, University of Michigan, Ann Arbor, MI, United States; Devin Peipert, Northwestern University The Feinberg School of Medicine, Chicago, IL, United States

Presenting Author(s)

Eloise Salmon, MD

Clinical Assistant Professor
University of Michigan Medical School
Ann Arbor, Michigan, United States

Background:

Children with nephrotic syndrome (NS) experience fluid overload (FO) which can impact health-related quality of life (HRQOL). In partnership with the Food and Drug Administration (FDA), PREPARing a clinical outcomes assessment set for Nephrotic Syndrome (Prepare-NS) aims to create and validate clinical outcome assessments (COAs) of FO in NS for use in drug development. For young children, the COA will be an observer reported outcome (ObsRO) from family caregivers. ObsROs are measures that reflect observable signs, behaviors, or events that can only be captured by observation in daily life (outside of a healthcare setting) by someone other than the patient or health care professional.

Objective:

To develop a conceptual model that captures important aspects of fluid overload and its impact on functioning that are observable by family caregivers for children ages 2-11 years with NS.

Design/Methods:

Parents/guardians caring for a child (aged 2-11) with FSGS, Minimal Change Disease, IgM Nephropathy, Membranous Nephropathy, or childhood-onset nephrotic syndrome not biopsied who has current or history of NS-associated edema within the past 3 months volunteered via study website to complete an interview. Concept elicitation interviews occurred via Zoom or phone and discussed how the child’s “NS” impacts functioning, with a specific focus on observable experiences. Interview transcripts were coded in NVivo by two independent raters according to established standards for qualitative analysis. Recurring themes informed our conceptual model.

Results:

Twenty-four care givers of children with nephrotic syndrome completed interviews. Image 1 shows demographics of the affected children. Themes overall were consistent among age, sex, race, and diagnostic subgroups. Image 2 shows the conceptual model.

Conclusion(s):

FO impacts the HRQOL for children in the domains of physical function, fatigue, sleep disturbance, gastrointestinal symptoms, appetite loss, and other essential functions. The next phase of Prepare-NS will be to develop the ObsRO inclusive of items to assess the impact of FO that is likely to be modifiable in disease-modifying drug development for young children with NS.