Frontiers in Medicine (Jul 2022)
Development and Implementation of the AIDA International Registry for Patients With VEXAS Syndrome
- Antonio Vitale,
- Valeria Caggiano,
- Francesca Della Casa,
- José Hernández-Rodríguez,
- Micol Frassi,
- Sara Monti,
- Sara Monti,
- Abdurrahman Tufan,
- Salvatore Telesca,
- Edoardo Conticini,
- Gaafar Ragab,
- Gaafar Ragab,
- Giuseppe Lopalco,
- Ibrahim Almaghlouth,
- Ibrahim Almaghlouth,
- Rosa Maria R. Pereira,
- Derya Yildirim,
- Marco Cattalini,
- Achille Marino,
- Teresa Giani,
- Francesco La Torre,
- Piero Ruscitti,
- Emma Aragona,
- Ewa Wiesik-Szewczyk,
- Emanuela Del Giudice,
- Petros P. Sfikakis,
- Marcello Govoni,
- Giacomo Emmi,
- Maria Cristina Maggio,
- Roberto Giacomelli,
- Francesco Ciccia,
- Giovanni Conti,
- Djouher Ait-Idir,
- Claudia Lomater,
- Vito Sabato,
- Matteo Piga,
- Ali Sahin,
- Daniela Opris-Belinski,
- Ruxandra Ionescu,
- Elena Bartoloni,
- Franco Franceschini,
- Paola Parronchi,
- Amato de Paulis,
- Amato de Paulis,
- Gerard Espinosa,
- Armin Maier,
- Gian Domenico Sebastiani,
- Antonella Insalaco,
- Farhad Shahram,
- Paolo Sfriso,
- Francesca Minoia,
- Maria Alessio,
- Joanna Makowska,
- Gülen Hatemi,
- Nurullah Akkoç,
- Francesca Li Gobbi,
- Antonio Gidaro,
- Alma Nunzia Olivieri,
- Sulaiman M. Al-Mayouf,
- Sükran Erten,
- Stefano Gentileschi,
- Ibrahim Vasi,
- Maria Tarsia,
- Ayman Abdel-Monem Ahmed Mahmoud,
- Bruno Frediani,
- Musa Fares Alzahrani,
- Ahmed Hatem Laymouna,
- Francesca Ricci,
- Fabio Cardinale,
- Karina Jahnz-Rózyk,
- Gian Marco Tosi,
- Francesca Crisafulli,
- Alberto Balistreri,
- Marília A. Dagostin,
- Mahmoud Ghanema,
- Carla Gaggiano,
- Jurgen Sota,
- Ilenia Di Cola,
- Claudia Fabiani,
- Henrique A. Mayrink Giardini,
- Alessandra Renieri,
- Alessandra Renieri,
- Alessandra Renieri,
- Alessandra Fabbiani,
- Alessandra Fabbiani,
- Alessandra Fabbiani,
- Anna Carrer,
- Anna Carrer,
- Anna Carrer,
- Monica Bocchia,
- Federico Caroni,
- Donato Rigante,
- Donato Rigante,
- Luca Cantarini
Affiliations
- Antonio Vitale
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Valeria Caggiano
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Francesca Della Casa
- Section of Clinical Immunology, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy
- José Hernández-Rodríguez
- Vasculitis Research Unit and Autoinflammatory Diseases Clinical Unit, Department of Autoimmune Diseases, Hospital Clinic of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Barcelona, Spain
- Micol Frassi
- Rheumatology and Clinical Immunology Unit, Department of Clinical and Experimental Sciences, ASST Spedali Civili, University of Brescia, Brescia, Italy
- Sara Monti
- Rheumatology Department, IRCCS Policlinico S. Matteo Foundation, Pavia, Italy
- Sara Monti
- Department of Experimental Medicine, University of Pavia, Pavia, Italy
- Abdurrahman Tufan
- Department of Internal Medicine and Rheumatology, Gazi University, Ankara, Turkey
- Salvatore Telesca
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Edoardo Conticini
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Gaafar Ragab
- Rheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt
- Gaafar Ragab
- Faculty of Medicine, Newgiza University (NGU), Giza, Egypt
- Giuseppe Lopalco
- 0Rheumatology Unit, Department of Emergency and Organ Transplantation, University of Bari, Bari, Italy
- Ibrahim Almaghlouth
- 1Rheumatology Unit, Department of Medicine, College of Medicine, King Saud University, Riyadh, Saudi Arabia
- Ibrahim Almaghlouth
- 2College of Medicine Research Center, College of Medicine, King Saud University, Riyadh, Saudi Arabia
- Rosa Maria R. Pereira
- 3Rheumatology Division, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, Brazil
- Derya Yildirim
- Department of Internal Medicine and Rheumatology, Gazi University, Ankara, Turkey
- Marco Cattalini
- 4Pediatric Clinic, Spedali Civili di Brescia, University of Brescia, Brescia, Italy
- Achille Marino
- 5Unit of Pediatric Rheumatology, ASST Gaetano Pini-CTO, Milan, Italy
- Teresa Giani
- 6Department of Clinical Sciences and Community Health, Research Center for Adult and Pediatric Rheumatic Diseases, ASST G. Pini-CTO, University of Milan, Milan, Italy
- Francesco La Torre
- 7Department of Pediatrics, Pediatric Rheumatology Center, Ospedale “Giovanni XXIII”, Azienda Ospedaliera-Universitaria Consorziale Policlinico, Bari, Italy
- Piero Ruscitti
- 8Rheumatology Unit, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy
- Emma Aragona
- 9Division of Gastroenterology, Ospedali Riuniti Villa Sofia-Vincenzo Cervello, Palermo, Italy
- Ewa Wiesik-Szewczyk
- 0Department of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defence, Military Institute of Medicine, Warsaw, Poland
- Emanuela Del Giudice
- 1Department of Maternal Infantile and Urological Sciences, Polo Pontino, Sapienza University of Rome, Rome, Italy
- Petros P. Sfikakis
- 2Joint Academic Rheumatology Program, 1st Department of Propedeutic Internal Medicine, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece
- Marcello Govoni
- 3Rheumatology Unit, Department of Medical Sciences, Azienda Ospedaliero-Universitaria S. Anna–Ferrara, University of Ferrara, Ferrara, Italy
- Giacomo Emmi
- 4Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
- Maria Cristina Maggio
- 5University Department Pro.Sa.M.I. “G. D'Alessandro”, University of Palermo, Palermo, Italy
- Roberto Giacomelli
- 6Rheumatology, Immunology and Clinical Medicine Unit, Department of Medicine, Università Campus Bio-Medico di Roma, Selcetta, Italy
- Francesco Ciccia
- 7Department of Precision Medicine, Università Degli Studi Della Campania Luigi Vanvitelli, Naples, Italy
- Giovanni Conti
- 8Pediatric Nephrology and Rheumatology Unit, AOU G Martino, Messina, Italy
- Djouher Ait-Idir
- 9Research Laboratory, Biodiversity, Biotechnology, Environment and Sustainable Development, Faculty of Sciences, M'Hamed Bougara University, Boumerdes, Algeria
- Claudia Lomater
- 0Unità Operativa (UO) Reumatologia, AO Ordine Mauriziano, Turin, Italy
- Vito Sabato
- 1Department of Immunology, Allergology, and Rheumatology, Antwerp University Hospital, University of Antwerp, Antwerp, Belgium
- Matteo Piga
- 2Rheumatology Unit, Department of Medical Sciences, University and Azienda Ospedaliera-Universitaria of Cagliari, Cagliari, Italy
- Ali Sahin
- 3Division of Rheumatology, Department of Internal Medicine, Sivas Cumhuriyet University, Sivas, Turkey
- Daniela Opris-Belinski
- 4Department of Internal Medicine and Rheumatology, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania
- Ruxandra Ionescu
- 4Department of Internal Medicine and Rheumatology, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania
- Elena Bartoloni
- 5Rheumatology Unit, Department of Medicine, University of Perugia, Perugia, Italy
- Franco Franceschini
- Rheumatology and Clinical Immunology Unit, Department of Clinical and Experimental Sciences, ASST Spedali Civili, University of Brescia, Brescia, Italy
- Paola Parronchi
- 4Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
- Amato de Paulis
- Section of Clinical Immunology, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy
- Amato de Paulis
- 6Center for Basic and Clinical Immunology Research (CISI), WAO Center of Excellence, University of Naples Federico II, Naples, Italy
- Gerard Espinosa
- Vasculitis Research Unit and Autoinflammatory Diseases Clinical Unit, Department of Autoimmune Diseases, Hospital Clinic of Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Barcelona, Spain
- Armin Maier
- 7Rheumatology Unit, Department of Medicine, Central Hospital of Bolzano, Bolzano, Italy
- Gian Domenico Sebastiani
- 8Unità Operativa Complessa (U.O.C.) Reumatologia, Ospedale San Camillo-Forlanini, Rome, Italy
- Antonella Insalaco
- 9Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, IRCCS (European Reference Network for Immunodeficiency, Autoinflammatory and Autoimmune Diseases Center), Rome, Italy
- Farhad Shahram
- 0Behcet's Disease Unit, Rheumatology Research Center, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran
- Paolo Sfriso
- 1Rheumatology Unit, Department of Medicine DIMED, University of Padova, Padova, Italy
- Francesca Minoia
- 2Pediatric Rheumatology, Fondazione IRCCS (Istituto di ricovero e cura a carattere scientifico) Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Maria Alessio
- 3Pediatric Rheumatology Unit, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy
- Joanna Makowska
- 4Department of Rheumatology, Medical University of Lodz, Lodz, Poland
- Gülen Hatemi
- 5Division of Rheumatology, Department of Internal Medicine, Behçet's Disease Research Center, Istanbul University–Cerrahpasa, Istanbul, Turkey
- Nurullah Akkoç
- 6Division of Rheumatology, Department of Internal Medicine, School of Medicine, Manisa Celal Bayar University, Manisa, Turkey
- Francesca Li Gobbi
- 7Rheumatology Unit, San Giovanni di Dio Hospital, Firenze, Italy
- Antonio Gidaro
- 8Department of Biomedical and Clinical Sciences 'Luigi Sacco', University of Milan, Milan, Italy
- Alma Nunzia Olivieri
- 9Department of Woman, Child and of General and Specialized Surgery, University of Campania “Luigi Vanvitelli”, Naples, Italy
- Sulaiman M. Al-Mayouf
- 0King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
- Sükran Erten
- 1Department of Rheumatology, Ankara City Hospital, Ankara, Turkey
- Stefano Gentileschi
- 2Unit of Rheumatology, Azienda Ospedaliero-Universitaria Senese, Siena, Italy
- Ibrahim Vasi
- Department of Internal Medicine and Rheumatology, Gazi University, Ankara, Turkey
- Maria Tarsia
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Ayman Abdel-Monem Ahmed Mahmoud
- Rheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt
- Bruno Frediani
- 2Unit of Rheumatology, Azienda Ospedaliero-Universitaria Senese, Siena, Italy
- Musa Fares Alzahrani
- 3Department of Medicine, College of Medicine, King Saud University, Riyadh, Saudi Arabia
- Ahmed Hatem Laymouna
- Rheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt
- Francesca Ricci
- 4Pediatric Clinic, Spedali Civili di Brescia, University of Brescia, Brescia, Italy
- Fabio Cardinale
- 7Department of Pediatrics, Pediatric Rheumatology Center, Ospedale “Giovanni XXIII”, Azienda Ospedaliera-Universitaria Consorziale Policlinico, Bari, Italy
- Karina Jahnz-Rózyk
- 0Department of Internal Medicine, Pulmonology, Allergy and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defence, Military Institute of Medicine, Warsaw, Poland
- Gian Marco Tosi
- 4Ophthalmology Unit, Department of Medicine, Surgery and Neurosciences, University of Siena, Siena, Italy
- Francesca Crisafulli
- Rheumatology and Clinical Immunology Unit, Department of Clinical and Experimental Sciences, ASST Spedali Civili, University of Brescia, Brescia, Italy
- Alberto Balistreri
- 5Bioengineering and Biomedical Data Science Lab, Department of Medical Biotechnologies, University of Siena, Siena, Italy
- Marília A. Dagostin
- 3Rheumatology Division, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, Brazil
- Mahmoud Ghanema
- Rheumatology and Clinical Immunology Unit, Internal Medicine Department, Faculty of Medicine, Cairo University, Giza, Egypt
- Carla Gaggiano
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Jurgen Sota
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- Ilenia Di Cola
- 8Rheumatology Unit, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy
- Claudia Fabiani
- 4Ophthalmology Unit, Department of Medicine, Surgery and Neurosciences, University of Siena, Siena, Italy
- Henrique A. Mayrink Giardini
- 3Rheumatology Division, Faculdade de Medicina, Hospital das Clínicas HCFMUSP, Universidade de São Paulo, São Paulo, Brazil
- Alessandra Renieri
- 6Medical Genetics, Department of Medical Biotechnologies, University of Siena, Siena, Italy
- Alessandra Renieri
- 7Department of Medical Biotechnologies, Med Biotech Hub and Competence Center, University of Siena, Siena, Italy
- Alessandra Renieri
- 8Genetica Medica, Azienda Ospedaliero-Universitaria Senese, Siena, Italy
- Alessandra Fabbiani
- 6Medical Genetics, Department of Medical Biotechnologies, University of Siena, Siena, Italy
- Alessandra Fabbiani
- 7Department of Medical Biotechnologies, Med Biotech Hub and Competence Center, University of Siena, Siena, Italy
- Alessandra Fabbiani
- 8Genetica Medica, Azienda Ospedaliero-Universitaria Senese, Siena, Italy
- Anna Carrer
- 6Medical Genetics, Department of Medical Biotechnologies, University of Siena, Siena, Italy
- Anna Carrer
- 7Department of Medical Biotechnologies, Med Biotech Hub and Competence Center, University of Siena, Siena, Italy
- Anna Carrer
- 8Genetica Medica, Azienda Ospedaliero-Universitaria Senese, Siena, Italy
- Monica Bocchia
- 9Unit of Hematology, Azienda Ospedaliera Universitaria Senese, University of Siena, Siena, Italy
- Federico Caroni
- 9Unit of Hematology, Azienda Ospedaliera Universitaria Senese, University of Siena, Siena, Italy
- Donato Rigante
- 0Department of Life Sciences and Global Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy
- Donato Rigante
- 1Rare Diseases and Periodic Fevers Research Centre, Università Cattolica del Sacro Cuore, Rome, Italy
- Luca Cantarini
- Department of Medical Sciences, Surgery and Neurosciences, Research Center of Systemic Autoinflammatory Diseases and Behçet's Disease Clinic, University of Siena, Siena, Italy
- DOI
- https://doi.org/10.3389/fmed.2022.926500
- Journal volume & issue
-
Vol. 9
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.
Keywords
