Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome

Abstract

ObjectiveThe aim of this paper is to present the AutoInflammatory Disease Alliance (AIDA) international Registry dedicated to Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome, describing its design, construction, and modalities of dissemination.MethodsThis Registry is a clinical, physician-driven, population- and electronic-based instrument designed for the retrospective and prospective collection of real-life data. Data gathering is based on the Research Electronic Data Capture (REDCap) tool and is intended to obtain real-world evidence for daily patients' management. The Registry may potentially communicate with other on-line tools dedicated to VEXAS syndrome, thus enhancing international collaboration and data sharing for research purposes. The Registry is practical enough to be easily modified to meet future needs regarding VEXAS syndrome.ResultsTo date (April 22nd, 2022), 113 Centers from 23 Countries in 4 continents have been involved; 324 users (114 Principal Investigators, 205 Site Investigators, 2 Lead Investigators, and 3 data managers) are currently able to access the registry for data entry (or data sharing) and collection. The Registry includes 4,952 fields organized into 18 instruments designed to fully describe patient's details about demographics, clinical manifestations, symptoms, histologic details about skin and bone marrow biopsies and aspirate, laboratory features, complications, comorbidities, therapies, and healthcare access.ConclusionThis international Registry for patients with VEXAS syndrome will allow the achievement of a comprehensive knowledge about this new disease, with the final goal to obtain real-world evidence for daily clinical practice, especially in relation to the comprehension of this disease about the natural history and the possible therapeutic approaches. This Project can be found on https://clinicaltrials.gov NCT05200715.

VEXAS syndrome: a new paradigm for adult-onset monogenic autoinflammatory diseases

“…In this regard, it is useful to join forces internationally and collect sufficient data and obtain solid and definitive results. To this end, the international registry dedicated to VEXAS syndrome, supported by the AutoInflammatory Disease Alliance (AIDA) Network, has been developed and is already enrolling patients [ 46 ].…”

A case of VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) with decreased oxidative stress levels after oral prednisone and tocilizumab treatment

“…Of course, it is difficult to conclude that d-ROM is an indicator of disease activity in VEXAS syndrome based on only this one case. The AutoInflammatory Disease Alliance (AIDA) keeps an international registry that includes VEXAS syndrome cases ( 19 ). We hope that further studies will be planned.…”

VEXAS: where do we stand 2 years later?

“…However, it seems very important to get an expert consensus on response criteria to harmonize the reports coming from all over the world and generate high-quality data. An initiative to build such a consensus has been launched by the EuroBloodNet collaborative network, and others have proposed an international registry of VEXAS patients [28]. To contribute to these efforts, we would like to propose response criteria for discussion (Fig.…”