Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies
Abstract Background Patient reported outcome (PRO) instruments are important for assessing the effects of new therapeutics; however, no PRO measures have been specifically developed for use in Nephrotic Syndrome (NS) with documented evidence of content validity. Methodology The Nephrotic Syndrome Sy...
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| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
SpringerOpen
2025-08-01
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| Series: | Journal of Patient-Reported Outcomes |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s41687-025-00937-7 |
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| Summary: | Abstract Background Patient reported outcome (PRO) instruments are important for assessing the effects of new therapeutics; however, no PRO measures have been specifically developed for use in Nephrotic Syndrome (NS) with documented evidence of content validity. Methodology The Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) tool was developed following a literature review, concept elicitation (CE) interviews with patients with NS and clinicians, item generation, and cognitive debriefing (CD) interviews with patients with NS. The CE interviews were conducted with four nephrologists and among adults (aged ≥ 18 years) and adolescents (aged 12–17 years) diagnosed with NS. Results In total, 45 adult and adolescent patients were interviewed across all CE and CD interviews. In the adult CE interviews, the most frequently reported symptoms were swelling in the feet/legs (n = 14/16), general tiredness (n = 13/16), change in appetite (n = 13/16), foamy urine (n = 12/16), and shortness of breath (n = 12/16); impacts included emotional health, physical function limitations, sleep difficulties, and social/lifestyle limitations (n = 13/16). In adolescents, the most frequently reported symptoms were tiredness/fatigue (n = 13/15), stomachache (n = 11/15), headache (n = 8/15), foamy/frothy urine (n = 7/15), swelling in face/eyes (n = 6/15), swelling in legs/ankles (n = 5/15), and change in appetite (n = 5/15), while the most common impacts were impact on schooling (n = 10/15), reduced ability to practice sports (n = 7/15), and impact on friendship (n = 7/15). A conceptual model was developed and used to inform item generation for the NephroSSI-PRO. During CD interviews, there were no challenges in understanding and relating to instrument instructions, items, and response options. Conclusions We have developed a PRO instrument to evaluate the symptoms and impacts of NS in adult and adolescent patients. In this study, the evidence from a literature review, and direct patient input from CE and CD support its content validity. Further work supporting the instrument’s validity will be performed in a future clinical trial. |
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| ISSN: | 2509-8020 |