Introduction: Fluid overload is a source of substantial morbidity for adults and children with nephrotic syndrome (NS). Preparation and Rationale for a Fluid Overload in Nephrotic Syndrome Clinical Outcomes Assessment Set for Drug Development (Prepare-NS, 5UG3FD007308) was funded by the US Food and Drug Administration to develop a core set of patient-reported and observer-reported (for young children) outcome measures of fluid overload for use in pharmaceutical trials across the lifespan.
Methods: The Prepare-NS study team developed the proposed context of use with input from stakeholders.
Interpretation of patient-reported outcome (PRO) scores has been supported by identifying score thresholds or ranges that indicate clinical importance. There has been a recent focus on the estimation of meaningful within patient change (MWPC). While much attention has been focused on anchor-based methods, some researchers prefer that a lower bound to these estimates should exceed a change score that could be observed due to measurement error alone as a safeguard against misclassifying individual patients as changed when they have not.
View Article and Find Full Text PDFBackground: The authors examined baseline physical functional (PF) impairment among cancer outpatients in the National Cancer Institute Cancer Moonshot study Northwestern University Improving the Management of Symptoms During and Following Cancer Treatment (NU IMPACT). They hypothesized that PF impairment, measured with the Patient Reported Outcome Measurement Information System-Physical Function (PROMIS-PF) survey, would (1) be common and more prevalent for patients receiving treatment compared with no treatment and (2) differ across tumor types, independent of cancer continuum phase.
Methods: Adults who were diagnosed with cancer in NU IMPACT (n = 2273) were sampled, and their PROMIS-PF scores were compared across tumor types and cancer continuum (curative, noncurative, or no treatment), with scores ≤40 indicating moderate-severe impairment.