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
Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy.
Section of Clinical Immunology, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.
Front Med (Lausanne). 2022 Jul 11;9:926500. doi: 10.3389/fmed.2022.926500. eCollection 2022.
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.
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.
To date (April 22, 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.
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.
本文旨在介绍致力于空泡、E1酶、X连锁、自身炎症性、体细胞（VEXAS）综合征的自身炎症性疾病联盟（AIDA）国际注册库，描述其设计、构建及传播方式。
该注册库是一种临床的、由医生主导的、基于人群和电子的工具，旨在回顾性和前瞻性地收集实际数据。数据收集基于研究电子数据采集（REDCap）工具，旨在获取日常患者管理的真实世界证据。该注册库可能会与其他致力于VEXAS综合征的在线工具进行交流，从而加强国际合作与数据共享以用于研究目的。该注册库实用性强，可轻松修改以满足未来关于VEXAS综合征的需求。
截至（2022年4月22日），来自四大洲23个国家的113个中心参与其中；目前有324名用户（114名主要研究者、205名现场研究者、2名首席研究者和3名数据管理员）能够访问该注册库进行数据录入（或数据共享）和收集。该注册库包括4952个字段，分为18种工具，旨在全面描述患者的人口统计学、临床表现、症状、皮肤和骨髓活检及抽吸物的组织学细节、实验室特征、并发症、合并症、治疗方法以及医疗保健获取等详细信息。
这个针对VEXAS综合征患者的国际注册库将有助于全面了解这种新疾病，最终目标是获取日常临床实践的真实世界证据，特别是在理解该疾病的自然史和可能的治疗方法方面。该项目可在https://clinicaltrials.gov NCT05200715上找到。
