JMIR Publications

ABSTRACT

Background:

The conduct of clinical trials in rare diseases utilizing the traditional centers of excellence approach 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 online registry cohort of participants with a self-reported diagnosis of GPA or MPA.

Methods:

Data was collected through standardized questionnaires among patients with a self-reported diagnosis of GPA or MPA in the Vasculitis Patient-Powered Research Network, a prospective, longitudinal cohort in which participants participate in an online platform.

Results:

There were 762 and 164 participants included with a self-diagnosis of GPA and MPA, respectively, with 98% of diagnoses confirmed by a physician. Compared to MPA, patients with GPA reported more ear, nose, and throat manifestations (including nasal/sinus, hearing loss and tracheal involvement; P<0.001 in each), fevers (P=0.050), joint involvement (P=0.003) and pulmonary involvement (P=0.002). Participants with MPA reported more renal involvement (P<0.001) and renal transplantation (P=0.015). ANCA positivity was reported in 94% (GPA) and 96% (MPA), and a biopsy showing vasculitis in 77% (GPA) and 82% (MPA).

Conclusions:

In this large online cohort of patients with a self-reported diagnosis of GPA or MPA, disease manifestations were consistent with what would be expected for each type of ANCA-associated vasculitis. Given the rarity of these and other vasculitides, conducting some types of research through online registries may provide an efficient alternative to in-person, center-of-excellence clinical trials.