Preparation and Rationale for a Patient-Centered Clinical Outcome Assessment Set of Fluid Overload for Drug...

Area of Science:

• Nephrology
• Clinical Outcomes Assessment
• Drug Development

Background:

• Fluid overload is a major complication in nephrotic syndrome (NS), affecting both pediatric and adult patients.
• Existing patient-reported outcome measures for NS are often nonspecific and do not adequately capture the impact of fluid overload.

Purpose of the Study:

• To develop a core set of patient- and observer-reported outcome measures for fluid overload in nephrotic syndrome.
• To support pharmaceutical trials for drug development in NS across the lifespan.

Main Methods:

• A scoping review of existing literature on patient- and observer-reported measures for fluid overload in NS was conducted.
• Secondary analyses of existing qualitative and quantitative data were performed.
• Stakeholder input was gathered to define the context of use.

Main Results:

• The outcome set is intended for individuals aged 2 and older with primary NS conditions, including FSGS, MCD, IgM nephropathy, and membranous nephropathy.
• Current literature shows a reliance on general health-related quality of life measures, with fluid overload linked to poorer scores.

Conclusions:

• The Prepare-NS study is addressing the need for specific fluid overload measures in NS.
• Qualitative studies are underway for concept elicitation to inform new measure development, followed by psychometric evaluation and validation.