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RESEARCH PRODUCT
Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome
Antonio VitaleValeria CaggianoFrancesca Della CasaJosé Hernández-rodríguezMicol FrassiSara MontiAbdurrahman TufanSalvatore TelescaEdoardo ConticiniGaafar RagabGiuseppe LopalcoIbrahim AlmaghlouthRosa Maria R. PereiraDerya YildirimMarco CattaliniAchille MarinoTeresa GianiFrancesco La TorrePiero RuscittiEmma AragonaEwa Wiesik-szewczykEmanuela Del GiudicePetros P. SfikakisMarcello GovoniGiacomo EmmiMaria Cristina MaggioRoberto GiacomelliFrancesco CicciaGiovanni ContiDjouher Ait-idirClaudia LomaterVito SabatoMatteo PigaAli SahinDaniela Opris-belinskiRuxandra IonescuElena BartoloniFranco FranceschiniPaola ParronchiAmato De PaulisGerard EspinosaArmin MaierGian Domenico SebastianiAntonella InsalacoFarhad ShahramPaolo SfrisoFrancesca MinoiaMaria AlessioJoanna MakowskaGülen HatemiNurullah AkkoçFrancesca Li GobbiAntonio GidaroAlma Nunzia OlivieriSulaiman M. Al-mayoufSükran ErtenStefano GentileschiIbrahim VasiMaria TarsiaAyman Abdel-monem Ahmed MahmoudBruno FredianiMusa Fares AlzahraniAhmed Hatem LaymounaFrancesca RicciFabio CardinaleKarina Jahnz-rózykGian Marco TosiFrancesca CrisafulliAlberto BalistreriMarília A. DagostinMahmoud GhanemaCarla GaggianoJurgen SotaIlenia Di ColaClaudia FabianiHenrique A. Mayrink GiardiniAlessandra RenieriAlessandra FabbianiAnna CarrerMonica BocchiaFederico CaroniDonato RiganteLuca Cantarinisubject
RegistryKeywords: autoinflammatory diseases; clinical management; precision medicine; rare diseases; research; treatment.Settore MED/16 - REUMATOLOGIAresearchtreatmentprecision medicinerare diseasesrare diseaseGeneral Medicineautoinflammatory diseasestreatment.Settore MED/38 - Pediatria Generale E Specialisticaautoinflammatory diseaseVEXAS syndromeclinical managementHuman medicineKeywords: autoinflammatory diseasesdescription
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.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2022-07-11 |