Frontiers in Medicine (Jun 2023)
A patient-driven registry on Behçet’s disease: the AIDA for patients pilot project
- Carla Gaggiano,
- Alessandra Del Bianco,
- Jurgen Sota,
- Stefano Gentileschi,
- Piero Ruscitti,
- Roberto Giacomelli,
- Matteo Piga,
- Francesca Crisafulli,
- Sara Monti,
- Giacomo Emmi,
- Giacomo Emmi,
- Amato De Paulis,
- Amato De Paulis,
- Antonio Vitale,
- Maria Tarsia,
- Valeria Caggiano,
- Rossana Nuzzolese,
- Veronica Parretti,
- Claudia Fabiani,
- Giuseppe Lopalco,
- Armin Maier,
- Marco Cattalini,
- Donato Rigante,
- Donato Rigante,
- Marcello Govoni,
- Francesca Li Gobbi,
- Serena Guiducci,
- Paola Parronchi,
- Paola Parronchi,
- Achille Marino,
- Francesco Ciccia,
- Maria Cristina Maggio,
- Emma Aragona,
- Elena Bartoloni,
- Annamaria Iagnocco,
- Ombretta Viapiana,
- Gian Domenico Sebastiani,
- Silvana Guerriero,
- Antonella Insalaco,
- Emanuela Del Giudice,
- Giovanni Conti,
- Patrizia Barone,
- Alma Nunzia Olivieri,
- Antonio Brucato,
- Francesco Carubbi,
- Paola Triggianese,
- Angela Mauro,
- Gian Marco Tosi,
- Alex Fonollosa,
- Henrique Ayres Mayrink Giardini,
- Gaafar Ragab,
- Gaafar Ragab,
- Samar Tharwat,
- Samar Tharwat,
- José Hernández-Rodríguez,
- Petros P. Sfikakis,
- Katerina Laskari,
- Anastasios Karamanakos,
- Gerard Espinosa,
- Farhad Shahram,
- Haner Direskeneli,
- Andrea Hinojosa-Azaola,
- Daniela Opris-Belinski,
- Ibrahim A. AlMaghlouth,
- Ibrahim A. AlMaghlouth,
- Gülen Hatemi,
- Gülen Hatemi,
- Mehmet Akif Eksin,
- Fatos Önen,
- Ewa Więsik-Szewczyk,
- Nurullah Akkoç,
- Abdurrahman Tufan,
- Ali Şahin,
- Şükran Erten,
- Seza Ozen,
- Ezgi Deniz Batu,
- Bruno Frediani,
- Alberto Balistreri,
- Luca Cantarini
Affiliations
- Carla Gaggiano
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Alessandra Del Bianco
- S.I.M.B.A (Associazione Italiana Sindrome e Malattia di Behçet), Pontedera, Italy
- Jurgen Sota
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Stefano Gentileschi
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Piero Ruscitti
- Rheumatology Unit, Department of Biotechnological & Applied Clinical Sciences, University of L’Aquila, L’Aquila, Italy
- Roberto Giacomelli
- Rheumatology and Immunology Unit, Department of Medicine, University of Rome “Campus Biomedico”, Rome, Italy
- Matteo Piga
- Rheumatology Unit, Department of Medical Sciences, University and AOU of Cagliari, Cagliari, Italy
- Francesca Crisafulli
- Rheumatology and Clinical Immunology, Department of Clinical and Experimental Sciences, University of Brescia and Spedali Civili [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Brescia, Italy
- Sara Monti
- Division of Rheumatology, Fondazione IRCCS Policlinico San Matteo [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center], Pavia, Italy
- Giacomo Emmi
- Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
- Giacomo Emmi
- Centre for Inflammatory Diseases, Department of Medicine, Monash Medical Centre, Monash University, Clayton, VIC, Australia
- Amato De Paulis
- 0Department of Translational Medical Sciences, Section of Clinical Immunology, University of Naples Federico II, Naples, Italy
- Amato De Paulis
- 1Center for Basic and Clinical Immunology Research (CISI), WAO Center of Excellence, University of Naples Federico II, Naples, Italy
- Antonio Vitale
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Maria Tarsia
- 2Clinical Paediatrics, Department of Molecular Medicine and Development, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Valeria Caggiano
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Rossana Nuzzolese
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Veronica Parretti
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Claudia Fabiani
- 3Ophthalmology Unit, Department of Medicine, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Giuseppe Lopalco
- 4Rheumatology Unit, Department of Emergency and Organ Transplantation, University of Bari, Bari, Italy
- Armin Maier
- 5Rheumatology Unit, Department of Medicine, Central Hospital of Bolzano, Bolzano, Italy
- Marco Cattalini
- 6Pediatric Clinic, University of Brescia and Spedali Civili di Brescia [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Brescia, Italy
- Donato Rigante
- 7Department of Life Sciences and Global Health, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy
- Donato Rigante
- 8Rare Diseases and Periodic Fevers Research Centre, Università Cattolica del Sacro Cuore, Rome, Italy
- Marcello Govoni
- 9Rheumatology Unit, Department of Medical Sciences, Azienda Ospedaliero-Universitaria S. Anna-Ferrara, University of Ferrara, Ferrara, Italy
- Francesca Li Gobbi
- 0Rheumatology Unit, Hospital S. Giovanni di Dio, Azienda USL-Toscana Centro, Florence, Italy
- Serena Guiducci
- Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
- Paola Parronchi
- Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
- Paola Parronchi
- 1Immunology and Cellular Therapies Unit, University Hospital Careggi, Florence, Italy
- Achille Marino
- 2Unit of Pediatric Rheumatology, Azienda Socio-Sanitaria Territoriale (ASST) Gaetano Pini-Centro Specialistico Ortopedico Traumatologico (CTO), Milan, Italy
- Francesco Ciccia
- 3Department of Precision Medicine, Università Degli Studi Della Campania Luigi Vanvitelli, Naples, Italy
- Maria Cristina Maggio
- 4University Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (PROMISE) “G. D’Alessandro”, University of Palermo, Palermo, Italy
- Emma Aragona
- 5Division of Gastroenterology, Ospedali Riuniti Villa Sofia-Vincenzo Cervello, Palermo, Italy
- Elena Bartoloni
- 6Rheumatology Unit, Department of Medicine, University of Perugia, Perugia, Italy
- Annamaria Iagnocco
- 7Academic Rheumatology Center, Dipartimento Scienze Cliniche e Biologiche, Università degli Studi di Torino, Turin, Italy
- Ombretta Viapiana
- 8Rheumatology Unit, Department of Medicine, University of Verona, Verona, Italy
- Gian Domenico Sebastiani
- 9Rheumatology Unit, San Camillo – Forlanini Hospital, Rome, Italy
- Silvana Guerriero
- 0Department of Ophthalmology and Otolaryngology, University of Bari, Bari, Italy
- Antonella Insalaco
- 1Division of Rheumatology, Ospedale Pediatrico Bambino Gesù, IRCCS [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center], Rome, Italy
- Emanuela Del Giudice
- 2Pediatric and Neonatology Unit, Department of Maternal Infantile and Urological Sciences, Sapienza University of Rome, Latina, Italy
- Giovanni Conti
- 3Pediatric Nephrology and Rheumatology Unit, Azienda Ospedaliero Universitaria (AOU) G Martino, Messina, Italy
- Patrizia Barone
- 4Department of Clinical and Experimental Medicine, University of Catania, Catania, Italy
- Alma Nunzia Olivieri
- 5Department of Woman, Child and of General and Specialized Surgery, University of Campania “Luigi Vanvitelli”, Naples, Italy
- Antonio Brucato
- 6Department of Biomedical and Clinical Sciences, Fatebenefratelli Hospital, Università di Milano, Milan, Italy
- Francesco Carubbi
- 7Department of Life, Health & Environmental Sciences and Internal Medicine and Nephrology Unit, Department of Medicine, University of L’Aquila and ASL Avezzano-Sulmona-L’Aquila, San Salvatore Hospital, L’Aquila, Italy
- Paola Triggianese
- 8Rheumatology, Allergology and Clinical Immunology, Department of System Medicine, University of Rome Tor Vergata, Rome, Italy
- Angela Mauro
- 9Pediatric Rheumatology Unit, Department of Childhood and Developmental Medicine, Fatebenefratelli-Sacco Hospital, Milan, Italy
- Gian Marco Tosi
- 3Ophthalmology Unit, Department of Medicine, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Alex Fonollosa
- 0Department of Ophthalmology, Biocruces Bizkaia Health Research Institute, Cruces University Hospital, University of the Basque Country, Barakaldo, Spain
- Henrique Ayres Mayrink Giardini
- 1Rheumatology Division, Hospital das Clinicas (HCFMUSP), Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brazil
- Gaafar Ragab
- 2Rheumatology and Clinical Immunology Unit, Department of Internal Medicine, Faculty of Medicine, Cairo University, Giza, Egypt
- Gaafar Ragab
- 3Faculty of Medicine, New Giza University, Giza, Egypt
- Samar Tharwat
- 4Rheumatology and Immunology Unit, Internal Medicine Department, Mansoura University, Mansoura, Egypt
- Samar Tharwat
- 5Department of Internal Medicine, Faculty of Medicine, Horus University, New Damietta, Egypt
- José Hernández-Rodríguez
- 6Department of Autoimmune Diseases, Institut d’Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Hospital Clínic of Barcelona [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center], University of Barcelona, Barcelona, Spain
- Petros P. Sfikakis
- 7Joint Academic Rheumatology Program, Medical School, National and Kapodistrian University of Athens, Athens, Greece
- Katerina Laskari
- 7Joint Academic Rheumatology Program, Medical School, National and Kapodistrian University of Athens, Athens, Greece
- Anastasios Karamanakos
- 8Department of Rheumatology, Evangelismos General Hospital, Athens, Greece
- Gerard Espinosa
- 6Department of Autoimmune Diseases, Institut d’Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Hospital Clínic of Barcelona [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center], University of Barcelona, Barcelona, Spain
- Farhad Shahram
- 9Behcet’s Disease Unit, Rheumatology Research Center, Shariati Hospital Tehran University of Medical Sciences, Tehran, Iran
- Haner Direskeneli
- 0Department of Internal Medicine, Division of Rheumatology, Marmara University, Faculty of Medicine, Istanbul, Turkey
- Andrea Hinojosa-Azaola
- 1Department of Immunology and Rheumatology, Instituto Nacional de Ciencias Médicas Y Nutrición Salvador Zubirán, Mexico City, Mexico
- Daniela Opris-Belinski
- 2Rheumatology and Internal Medicine Department, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania
- Ibrahim A. AlMaghlouth
- 3Rheumatology Unit, Department of Medicine, College of Medicine, King Saud University, Riyadh, Saudi Arabia
- Ibrahim A. AlMaghlouth
- 4College of Medicine Research Center, College of Medicine, King Saud University, Riyadh, Saudi Arabia
- Gülen Hatemi
- 5Department of Internal Medicine, Division of Rheumatology, Cerrahpasa Medical School, Istanbul University-Cerrahpasa, Istanbul, Turkey
- Gülen Hatemi
- 6Behçet’s Disease Research Center, Istanbul University-Cerrahpasa, Istanbul, Turkey
- Mehmet Akif Eksin
- 7Rheumatology Clinic, Ankara City Hospital, Ankara, Turkey
- Fatos Önen
- 8Department of Internal Medicine Division of Rheumatology, Dokuz Eylül University Faculty of Medicine, İzmir, Turkey
- Ewa Więsik-Szewczyk
- 9Department of Internal Medicine, Pneumonology, Allergology and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defense, Military Institute of Medicine, National Research Institute, Warsaw, Poland
- Nurullah Akkoç
- 0Division of Rheumatology, Department of Internal Medicine, School of Medicine, Manisa Celal Bayar University, Manisa, Turkey
- Abdurrahman Tufan
- 1Division of Rheumatology, Department of Internal Medicine, Faculty of Medicine Gazi University, Ankara, Turkey
- Ali Şahin
- 2Division of Rheumatology, Department of Internal Medicine, Sivas Cumhuriyet University Medical Faculty, Sivas, Turkey
- Şükran Erten
- 7Rheumatology Clinic, Ankara City Hospital, Ankara, Turkey
- Seza Ozen
- 3Pediatric Rheumatology Unit, Department of Pediatrics, Hacettepe University School of Medicine, Ankara, Turkey
- Ezgi Deniz Batu
- 3Pediatric Rheumatology Unit, Department of Pediatrics, Hacettepe University School of Medicine, Ankara, Turkey
- Bruno Frediani
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- Alberto Balistreri
- 4Bioengineering and Biomedical Data Science Lab, Department of Medical Biotechnologies, University of Siena, Siena, Italy
- Luca Cantarini
- Rheumatology Unit, Department of Medical Sciences, Surgery and Neurosciences, University of Siena and Azienda Ospedaliero-Universitaria Senese [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory, and Autoimmune Diseases (RITA) Center], Siena, Italy
- DOI
- https://doi.org/10.3389/fmed.2023.1188021
- Journal volume & issue
-
Vol. 10
Abstract
IntroductionThis paper describes the creation and preliminary results of a patient-driven registry for the collection of patient-reported outcomes (PROs) and patient-reported experiences (PREs) in Behçet’s disease (BD).MethodsThe project was coordinated by the University of Siena and the Italian patient advocacy organization SIMBA (Associazione Italiana Sindrome e Malattia di Behçet), in the context of the AIDA (AutoInflammatory Diseases Alliance) Network programme. Quality of life, fatigue, socioeconomic impact of the disease and therapeutic adherence were selected as core domains to include in the registry.ResultsRespondents were reached via SIMBA communication channels in 167 cases (83.5%) and the AIDA Network affiliated clinical centers in 33 cases (16.5%). The median value of the Behçet’s Disease Quality of Life (BDQoL) score was 14 (IQR 11, range 0–30), indicating a medium quality of life, and the median Global Fatigue Index (GFI) was 38.7 (IQR 10.9, range 1–50), expressing a significant level of fatigue. The mean Beliefs about Medicines Questionnaire (BMQ) necessity-concern differential was 0.9 ± 1.1 (range – 1.8–4), showing that the registry participants prioritized necessity belief over concerns to a limited extent. As for the socioeconomic impact of BD, in 104 out of 187 cases (55.6%), patients had to pay from their own pocket for medical exams required to reach the diagnosis. The low family socioeconomic status (p < 0.001), the presence of any major organ involvement (p < 0.031), the presence of gastro-intestinal (p < 0.001), neurological (p = 0.012) and musculoskeletal (p = 0.022) symptoms, recurrent fever (p = 0.002), and headache (p < 0.001) were associated to a higher number of accesses to the healthcare system. Multiple linear regression showed that the BDQoL score could significantly predict the global socioeconomic impact of BD (F = 14.519, OR 1.162 [CI 0.557–1.766], p < 0.001).DiscussionPreliminary results from the AIDA for Patients BD registry were consistent with data available in the literature, confirming that PROs and PREs could be easily provided by the patient remotely to integrate physician-driven registries with complementary and reliable information.
Keywords
- Behçet’s disease
- patient-driven registries
- rare diseases
- autoinflammatory diseases
- patient involvement
- patient-reported outcomes
