Clinical Characteristics of an Internet-Based Cohort of Patient-Reported Diagnosis of Granulomatosis With Polyangiitis and Microscopic Polyangiitis: Observational Study

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Abstract

Background

Utilizing the traditional centers of excellence approach to conduct clinical trials involving rare diseases remains challenging. Patient-based registries have been shown to be both feasible and valid in several other diseases.

Objective

This report outlines the clinical characteristics of a large internet registry cohort of participants with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis.

Methods

Patients with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis in an internet-based prospective longitudinal cohort (from the Vasculitis Patient-Powered Research Network) were included. Data on symptoms, diagnostic testing, and treatment were collected using standardized questionnaires.

Results

The study compared patients with granulomatosis with polyangiitis (n=762) and patients with microscopic polyangiitis (n=164). Of the cohort, 97.7% (904/925) reported the diagnosis had been confirmed by a physician. Compared to microscopic polyangiitis, patients with granulomatosis with polyangiitis reported significantly more ear, nose, and throat manifestations (granulomatosis with polyangiitis: 641/723, 88.7%; microscopic polyangiitis: 89/164, 54.3%; z=10.42, P<.001), fevers (granulomatosis with polyangiitis: 325/588, 55.3%; microscopic polyangiitis: 64/139, 46.0%; z=1.96, P=.05), joint involvement (granulomatosis with polyangiitis: 549/688, 79.8%; microscopic polyangiitis: 106/154, 68.8%; z=2.96, P=.003), and pulmonary involvement (granulomatosis with polyangiitis: 523/734, 71.3%; microscopic polyangiitis: 90/154, 58.4%; z=3.13, P=.002). Compared to microscopic polyangiitis, patients with granulomatosis with polyangiitis reported significantly less renal involvement (granulomatosis with polyangiitis: 457/743, 61.5%; microscopic polyangiitis: 135/163, 82.8%; z=–5.18, P<.001) and renal transplantation (granulomatosis with polyangiitis: 10/721, 1.4%; microscopic polyangiitis: 7/164, 4.3%; z=–2.43, P=.02). Antineutrophil cytoplasmic antibody positivity was reported in 94.2% (652/692) of patients with granulomatosis with polyangiitis and 96.1% (147/153) of patients with microscopic polyangiitis. A biopsy showing vasculitis was reported in 77.0% (562/730) of patients with granulomatosis with polyangiitis and 81.9% (131/160) of patients with microscopic polyangiitis.

Conclusions

In this large, internet-based cohort of patients with a self-reported diagnosis of granulomatosis with polyangiitis or microscopic polyangiitis, disease manifestations were consistent with expectations for each type of vasculitis. Given the rarity of these and other vasculitides, conducting some types of research through internet-based registries may provide an efficient alternative to inperson, center-of-excellence clinical trials.

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Current standard therapy for Wegener's granulomatosis and microscopic polyangiitis combines corticosteroids and cyclophosphamide to induce remission, followed by a less toxic immunosuppressant such as azathioprine or methotrexate for maintenance therapy. However, azathioprine and methotrexate have not been compared with regard to safety and efficacy. In this prospective, open-label, multicenter trial, we randomly assigned patients with Wegener's granulomatosis or microscopic polyangiitis who entered remission with intravenous cyclophosphamide and corticosteroids to receive oral azathioprine (at a dose of 2.0 mg per kilogram of body weight per day) or methotrexate (at a dose of 0.3 mg per kilogram per week, progressively increased to 25 mg per week) for 12 months. The primary end point was an adverse event requiring discontinuation of the study drug or causing death; the sample size was calculated on the basis of the primary hypothesis that methotrexate would be less toxic than azathioprine. The secondary end points were severe adverse events and relapse. Among 159 eligible patients, 126 (79%) had a remission, were randomly assigned to receive a study drug in two groups of 63 patients each, and were followed for a mean (+/-SD) period of 29+/-13 months. Adverse events occurred in 29 azathioprine recipients and 35 methotrexate recipients (P=0.29); grade 3 or 4 events occurred in 5 patients in the azathioprine group and 11 patients in the methotrexate group (P=0.11). The primary end point was reached in 7 patients who received azathioprine as compared with 12 patients who received methotrexate (P=0.21), with a corresponding hazard ratio for methotrexate of 1.65 (95% confidence interval, 0.65 to 4.18; P=0.29). There was one death in the methotrexate group. Twenty-three patients who received azathioprine and 21 patients who received methotrexate had a relapse (P=0.71); 73% of these patients had a relapse after discontinuation of the study drug. These results do not support the primary hypothesis that methotrexate is safer than azathioprine. The two agents appear to be similar alternatives for maintenance therapy in patients with Wegener's granulomatosis and microscopic polyangiitis after initial remission. (ClinicalTrials.gov number, NCT00349674.) 2008 Massachusetts Medical Society

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Author and article information

Contributors

Jason Michael Springer:

ORCID: https://orcid.org/0000-0002-3903-6049

University of Kansas Medical Center3901 Rainbow Blvd MS 2026Kansas City, KS, 66160United States1 913 588 6009jspringer2@kumc.edu

Journal

Journal ID (nlm-ta): J Med Internet Res

Journal ID (iso-abbrev): J. Med. Internet Res

Journal ID (publisher-id): JMIR

Title: Journal of Medical Internet Research

Publisher: JMIR Publications (Toronto, Canada )

ISSN (Print): 1439-4456

ISSN (Electronic): 1438-8871

Publication date Collection: July 2020

Publication date (Electronic): 20 July 2020

Volume: 22

Issue: 7

Electronic Location Identifier: e17231

Affiliations

[1 ] University of Kansas Medical Center Kansas City, KS United States

[2 ] Division of Rheumatology Department of Medicine University of California Santa Monica, CA United States

[3 ] Division of Rheumatology Department of Medicine University of Pennsylvania Philadelphia, PA United States

[4 ] Vasculitis Foundation Kansas City, MO United States

[5 ] Health Informatics Institute University of South Florida Tampa, FL United States

[6 ] Division of Clinical Epidemiology Department of Biostatistics, Epidemiology, and Informatics University of Pennsylvania Philadelphia, PA United States

Author notes

Corresponding Author: Jason Michael Springer jspringer2@ 123456kumc.edu

Author information

Jason Michael Springer https://orcid.org/0000-0002-3903-6049

Tanaz A Kermani https://orcid.org/0000-0002-7335-7321

Antoine Sreih https://orcid.org/0000-0002-7864-0185

Dianne G Shaw https://orcid.org/0000-0002-2184-0266

Kalen Young https://orcid.org/0000-0002-2070-0894

Cristina M Burroughs https://orcid.org/0000-0002-0660-8127

Peter A Merkel https://orcid.org/0000-0001-9284-7345

Article

Publisher ID: v22i7e17231

DOI: 10.2196/17231

PMC ID: 7428147

PubMed ID: 32459634

SO-VID: 9d629377-62a2-44b3-82e7-f95c345e4696

Copyright © ©Jason Michael Springer, Tanaz A Kermani, Antoine Sreih, Dianne G Shaw, Kalen Young, Cristina M Burroughs, Peter A Merkel. Originally published in the Journal of Medical Internet Research (http://www.jmir.org), 20.07.2020.

License:

This is an open-access article distributed under the terms of the Creative Commons Attribution License ( https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work, first published in the Journal of Medical Internet Research, is properly cited. The complete bibliographic information, a link to the original publication on http://www.jmir.org/, as well as this copyright and license information must be included.

History

Date received : 24 February 2020

Date revision requested : 1 April 2020

Date revision received : 30 April 2020

Date accepted : 14 May 2020

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