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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2025-08-01
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| Series: | Journal of Patient-Reported Outcomes |
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| Online Access: | https://doi.org/10.1186/s41687-025-00937-7 |
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| author | Joshua Maher An-Qi Hu Sagar U. Nigwekar Julie Lin |
| author_facet | Joshua Maher An-Qi Hu Sagar U. Nigwekar Julie Lin |
| author_sort | Joshua Maher |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-aaac7cf9f78b4ea0ac8683216fc05484 |
| institution | Kabale University |
| issn | 2509-8020 |
| language | English |
| publishDate | 2025-08-01 |
| publisher | SpringerOpen |
| record_format | Article |
| series | Journal of Patient-Reported Outcomes |
| spelling | doaj-art-aaac7cf9f78b4ea0ac8683216fc054842025-08-24T11:32:39ZengSpringerOpenJournal of Patient-Reported Outcomes2509-80202025-08-019111310.1186/s41687-025-00937-7Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathiesJoshua Maher0An-Qi Hu1Sagar U. Nigwekar2Julie Lin3SanofiSanofiDivision of Nephrology, Department of Medicine, Massachusetts General BrighamSanofiAbstract 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.https://doi.org/10.1186/s41687-025-00937-7Clinical outcome assessmentsDisease impactsDisease symptomsHealth-related quality of lifeNephrotic syndromePatient-reported outcomes |
| spellingShingle | Joshua Maher An-Qi Hu Sagar U. Nigwekar Julie Lin Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies Journal of Patient-Reported Outcomes Clinical outcome assessments Disease impacts Disease symptoms Health-related quality of life Nephrotic syndrome Patient-reported outcomes |
| title | Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies |
| title_full | Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies |
| title_fullStr | Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies |
| title_full_unstemmed | Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies |
| title_short | Development of the Nephrotic Syndrome Symptom and Impact Patient Reported Outcome (NephroSSI-PRO) measure for use in adults and adolescents with primary glomerulopathies |
| title_sort | development of the nephrotic syndrome symptom and impact patient reported outcome nephrossi pro measure for use in adults and adolescents with primary glomerulopathies |
| topic | Clinical outcome assessments Disease impacts Disease symptoms Health-related quality of life Nephrotic syndrome Patient-reported outcomes |
| url | https://doi.org/10.1186/s41687-025-00937-7 |
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