Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome
Authors: Vitale Antonio , Caggiano Valeria , Della Casa Francesca , Hernández-Rodríguez José , Frassi Micol , Monti Sara , Tufan Abdurrahman , Telesca Salvatore , Conticini Edoardo , Ragab Gaafar , Lopalco Giuseppe , Almaghlouth Ibrahim , Pereira Rosa Maria R. , Yildirim Derya , Cattalini Marco , Marino Achille , Giani Teresa , La Torre Francesco , Ruscitti Piero , Aragona Emma , Wiesik-Szewczyk Ewa , Del Giudice Emanuela , Sfikakis Petros P. , Govoni Marcello , Emmi Giacomo , Maggio Maria Cristina , Giacomelli Roberto , Ciccia Francesco , Conti Giovanni , Ait-Idir Djouher , Lomater Claudia , Sabato Vito , Piga Matteo , Sahin Ali , Opris-Belinski Daniela , Ionescu Ruxandra , Bartoloni Elena , Franceschini Franco , Parronchi Paola , de Paulis Amato , Espinosa Gerard , Maier Armin , Sebastiani Gian Domenico , Insalaco Antonella , Shahram Farhad , Sfriso Paolo , Minoia Francesca , Alessio Maria , Makowska Joanna , Hatemi Gülen , Akkoç Nurullah , Li Gobbi Francesca , Gidaro Antonio , Olivieri Alma Nunzia , Al-Mayouf Sulaiman M. , Erten Sükran , Gentileschi Stefano , Vasi Ibrahim , Tarsia Maria , Mahmoud Ayman Abdel-Monem Ahmed , Frediani Bruno , Fares Alzahrani Musa , Laymouna Ahmed Hatem , Ricci Francesca , Cardinale Fabio , Jahnz-Rózyk Karina , Tosi Gian Marco , Crisafulli Francesca , Balistreri Alberto , Dagostin Marília A. , Ghanema Mahmoud , Gaggiano Carla , Sota Jurgen , Di Cola Ilenia , Fabiani Claudia , Giardini Henrique A. Mayrink , Renieri Alessandra , Fabbiani Alessandra , Carrer Anna , Bocchia Monica , Caroni Federico , Rigante Donato , Cantarini Luca 
Published: July 2022

Objective

The 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.

Methods

This 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.

Results

To 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.

Conclusion

This 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.