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Kidney Week

Abstract: FR-PO867

Patient-Reported Outcomes (PROs) Use in IgA Nephropathy (IgAN)

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

  • Richardson, Michelle M., Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
  • Ali, Sarah Naz, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
  • Gandhi, Hema Kannan, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
  • Oberdhan, Dorothee, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
Background

Immunoglobulin A Nephropathy (IgAN) is a progressive disease with high patient-reported burden and disease-related impact. New therapies could alter the course of disease, presenting the need for appropriate measurement of patient-reported outcomes (PROs). PRO requirements include content validity, acceptable measurement properties, information on meaningfulness and most importantly, must be fit for context of use (e.g., screening, diagnosis, likelihood of being impacted by treatment). With the advent of newer therapies, assessing PROs used in IgAN clinical trials is needed to evaluate their applicability for regulatory use.

Methods

We conducted a targeted MEDLINE search for PROs used in IgAN clinical trials from 2021-2024 to update a 2021 systematic review, compiled the PROs used and evaluated individual instrument characteristics: context of use, purpose and meaningful change over time.

Results

464 articles were identified, 11 remained after title and abstract review and 3 underwent full text review and were included. Of the 7 clinical trials (6 from the previous search, 1 newly identified), 1 was an interventional trial of the effects of exercise on depression symptoms, 1 was an observational longitudinal study, 2 were cross-sectional studies, and 3 were studies of PRO development work. Two studies enrolled only patients with IgAN, the others enrolled <10 IgAN patients each. 19 different PROs were used throughout the included studies: only 1 was specific to kidney disease (Peds QL ESRD module), 5 were generic measures, 14 were disease- or symptom-specific measures (e.g., depression, anxiety, mobility) 11 were for use in adults and 8 in pediatrics. No PRO met context of use or responsiveness criteria.

Conclusion

This review evaluated 19 PROs used in IgAN clinical trials. The only interventional longitudinal trial that was identified tested a life-style intervention and did not report responsiveness. One PRO was kidney-disease specific but was only used in pediatric patients. Currently, published studies of PROs in IgAN clinical trials do not provide sufficient information to support regulatory use and additional evidence is needed. In future research, selecting a high-quality PRO that is integrated with other clinical trial outcomes and most effective for regulatory use requires attention to context of use, purpose and meaningful change.

Funding

  • Commercial Support – Otsuka Pharmaceutical Development and Commercialization, Inc.