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. 2022 May 25;11(1):e27273.
doi: 10.2196/27273.

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

Irena Doubelt  1   2 , Jason M Springer  3 , Tanaz A Kermani  4 , Antoine G Sreih  5 , Cristina Burroughs  6 , David Cuthbertson  6 , Simon Carette  1   2 , Nader A Khalidi  7 , Curry L Koening  8 , Carol Langford  9 , Carol A McAlear  5 , Larry W Moreland  10 , Paul A Monach  11 , Dianne G Shaw  12 , Philip Seo  13 , Ulrich Specks  14 , Kenneth J Warrington  15 , Kalen Young  12 , Peter A Merkel  5 , Christian Pagnoux  1   2
Affiliations

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

Irena Doubelt et al. Interact J Med Res. .

Abstract

Background: Patient-based registries can help advance research on rare diseases such as eosinophilic granulomatosis with polyangiitis (EGPA), a complex multiorgan form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.

Objective: The aim of this study is to compare patient-reported and physician-reported data on manifestations, treatments, and outcomes for patients with EGPA.

Methods: We completed a comparative analysis of patients ≥18 years with EGPA in Canada and the United States from the following 2 cohorts: (1) The Vasculitis Patient-Powered Research Network (VPPRN), a self-enrolled secure portal with patient-entered data updated quarterly (2014-2019) and (2) the Vasculitis Clinical Research Consortium (VCRC) observational studies, a physician-entered database (2003-2019) of patients who fulfilled the 1990 American College of Rheumatology classification criteria for EGPA. The studied parameters included demographic characteristics, clinical manifestations, ANCA status, treatments, and relapses.

Results: Data from 195 patients with a validated diagnosis of EGPA in the VPPRN and 354 patients enrolled in the VCRC were analyzed. Compared to the VCRC cohort, the patients in the VPPRN cohort were more likely to be female (135/195, 69.2% compared to 209/354, 59%; P=.02) and younger at diagnosis (47.3 compared to 50.0 years; P=.03); both cohorts reported similar frequencies of asthma (177/184, 96.2% in the VPPRN cohort compared to 329/354, 92.9% in the VCRC cohort; P=.13) and cardiac manifestations (44/153, 28.8% compared to 75/354, 21.2%; P=.06), but the VPPRN cohort reported less frequent lung manifestations other than asthma and more frequent disease manifestations in all other organ systems. The ANCA positivity was 48.9% (64/131) in the VPPRN patients compared to 38.9% (123/316; P=.05) in the VCRC cohort. Relapsing disease after study enrollment was reported in 32.3% (63/195) of patients in the VPPRN compared to 35.7% (99/277) of patients in the VCRC. Most therapies (GC, cyclophosphamide, mepolizumab) were used at similar frequencies in both groups, except for rituximab with VPPRN patients reporting more use than the VCRC cohort (47/195, 24.1% compared to 29/277, 10.5%; P<.001).

Conclusions: Overall, patients and physicians report manifestations of EGPA at similar frequencies. However, observed differences between patient and physician reports imply the potential occurrence of selection biases. These results support the use of patient-reported data in EGPA but also the need for careful consideration of disease-specific definitions for the study of EGPA and how patient- and physician-reported data are collected.

Trial registration: ClinicalTrials.gov NCT00315380, https://clinicaltrials.gov/ct2/show/NCT00315380; ClinicalTrials.gov NCT01241305, https://clinicaltrials.gov/ct2/show/NCT01241305.

Keywords: clinical outcomes; digital health; eosinophilic granulomatosis with polyangiitis; granulomatosis; health databases; health network; patient outcomes; patient-reported outcomes measures; research network.

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Conflict of interest statement

Conflicts of Interest: CP reports receiving fees for serving on advisory boards for ChemoCentryx Inc, GlaxoSmithKline plc, Sanofi SA, F. Hoffman-La Roche AG, InflaRx GmbH, and AstraZeneca plc; he also reports receiving lecture fees and research grant support from F. Hoffman-La Roche AG and GlaxoSmithKline plc. PA Merkel reports receiving funds for the following activities in the past 2 years: consulting for AbbVie Inc, AstraZeneca plc, Biogen Inc, Boehringer Ingelheim, Bristol Myers Squibb Co, Celgene Corp, ChemoCentryx Inc, CSL Behring, Dynacure SA, EMD Serono, Forbius, Genentech Inc/F. Hoffman-La Roche AG, Genzyme Corp/Sanofi SA, GlaxoSmithKline plc, InflaRx GmbH, Insmed Inc, Janssen Inc, Kiniksa Pharmaceuticals Ltd, Kyverna Therapeutics Inc, Magenta Therapeutics Inc, Neutrolis Inc, Novartis AG, Pfizer Inc, Sparrow Pharmaceuticals Inc, Takeda Pharmaceutical Company Ltd, Talaris Therapeutics Inc; research support from AbbVie Inc, AstraZeneca plc, Boehringer Ingelheim, Bristol Myers Squibb Co, Celgene Corp, ChemoCentryx Inc, Eicos, Forbius, Genentech Inc/F. Hoffman-La Roche AG, Genzyme Corp/Sanofi SA, GlaxoSmithKline plc, InflaRx GmbH, Sanofi SA, and Takeda Pharmaceutical Company Ltd; and royalties from UpToDate Inc. AGS is an employee of Bristol Myers Squibb Co. NAK is part of the advisory board/speaker bureau at F. Hoffman-La Roche AG and also receives research grant support from F. Hoffman-La Roche AG, Bristol Myers Squibb Co, Sanofi, AbbVie Inc, and AstraZeneca plc.

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