PATIENT-LED DEVELOPMENT OF A PATIENT REPORTED OUTCOMES INSTRUMENT TO MEASURE HEALTH-RELATED QUALITY OF LIFE IN GLOMERULAR DISEASE (PRO-GD STUDY)

Glomerular diseases (GDs) are a leading cause of kidney failure worldwide. Biochemical (e.g., blood and urine test results) and clinical (e.g., kidney failure, death) outcomes are typically collected in GD research. However, patient-reported outcomes (PROs), which capture how a person feels and functions, are highly prioritized by patients, yet remain underreported in research due to a lack of disease-specific measure for the GD population. The PRO-GD study aims to address this unmet need by developing and validating a GD-specific PRO questionnaire. Here, we describe the study methodology and baseline characteristics of the cohort enrolled by 22 Oct 2025. 

PRO-GD is an ongoing, prospective, questionnaire-based cohort study conducted in Ireland. It aims to recruit 180 adults (≥18 years) with any of the following GDs who have not yet developed kidney failure: IgA nephropathy (IgAN), Minimal Change Disease, Focal Segmental Glomerulosclerosis (FSGS), Membranous Nephropathy, C3 Glomerulopathy, Membranoproliferative Glomerulonephritis, or frequently relapsing nephrotic syndrome since childhood. A prototype PRO-GD questionnaire was developed by selecting PROMIS (Patient-Reported Outcomes Measurement Information System) domains that closely align with the PROs identified as most important to patients with GD in prior qualitative research such as life participation, anxiety, and fatigue. This questionnaire was further refined in line with principles of Public and Patient Involvement (PPI), to include questions on medication impact and oedema. Socio-demographic, disease characteristics, treatment exposure, and comorbidity data are also collected. Patients submit data using the ‘Surveys’ module of an online electronic data capture (EDC) system (www.castoredc.com), or by returning a paper copy by post. Paper-based survey responses and GD-relevant blood and urine test results are entered manually to the EDC by the study team. Data analysis will include validation of the PRO-GD questionnaire (known-groups comparisons to determine construct validity; intraclass correlation coefficients to determine test-retest reliability), descriptive statistics to examine baseline characteristics, and generalized linear models to examine temporal changes in PRO scores (baseline to 6 months).

Recruitment began on Sep 1st, 2024, and will conclude by Dec 31st, 2025. By Oct 22, 2025, 80 participants were enrolled. The mean age was 50 (±16) years, and 46% were female. The most common diagnoses were IgAN (56%), Membranous Nephropathy (13%), Minimal Change Disease (9.7%), and FSGS (9.7%). Baseline PRO-GD questionnaire responses will evaluate HRQOL across multiple domains: we expect to have these data available for the fully recruited cohort by early 2026, in time for the WCN meeting. 

The PRO-GD study addresses an important clinical and research gap by developing a disease-specific PRO measure for patients with GD. Once internally and externally validated, the PRO-GD questionnaire can be applied in clinical and research practice, enabling treatment effectiveness to be evaluated from the patient’s perspective and ensuring that the patient voice is heard.