Self-Reported Data and Physician-Reported Data in Patients With Eosinophilic Granulomatosis With Polyangiitis:...

Area of Science:

• Rheumatology
• Rare Diseases
• Patient-Reported Outcomes

Background:

• Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare, complex, multi-organ vasculitis associated with antineutrophil cytoplasmic antibodies (ANCA).
• Patient registries are crucial for advancing research in rare diseases like EGPA.

Purpose of the Study:

• To compare patient-reported versus physician-reported data on EGPA manifestations, treatments, and outcomes.
• To identify potential discrepancies and biases between patient and physician data collection methods.

Main Methods:

• Comparative analysis of two cohorts: Vasculitis Patient-Powered Research Network (VPPRN) with patient-entered data and Vasculitis Clinical Research Consortium (VCRC) with physician-entered data.
• Inclusion of adult patients (≥18 years) with EGPA diagnosed using 1990 ACR criteria.
• Analysis of demographic characteristics, clinical manifestations, ANCA status, treatments, and relapse rates.

Main Results:

• VPPRN cohort had more females and younger patients at diagnosis compared to VCRC.
• Similar frequencies of asthma and cardiac involvement; VPPRN reported fewer lung issues (excluding asthma) but more manifestations in other organs.
• Higher ANCA positivity in VPPRN (48.9%) vs. VCRC (38.9%); similar relapse rates.
• Rituximab use was significantly higher in the VPPRN cohort.

Conclusions:

• Patient and physician reports on EGPA manifestations are generally consistent.
• Discrepancies highlight potential selection biases in data collection.
• Supports the utility of patient-reported outcomes in EGPA research while emphasizing the need for standardized definitions and careful data interpretation.